Archive for January, 2020
Osaka University-based team successfully conducts first transplantation of cardiac muscle cells around the globe – Medical Herald
A team based at Osaka University stated how it had succeeded in carrying out the first transplant of cardiac muscle cells, around the globe, developed from iPS cells in a clinical trial which as physician-initiated.
A professor in Osaka Universitys cardiovascular surgery unit, Yoshiki Sawa, along with his colleagues at the university, intend to transplant heart muscle cell sheets into 10 individuals experiencing severe heart malfunction as a result of ischemic cardiomyopathy, in a clinical trial, to validate the safety and the effectiveness of the therapy with the use of induced pluripotent stem cells.
On the surface of the hearts of the partaking individuals, the cells on the degradable sheets are attached. It is predicted that these cells will develop to release a protein that can allow for the regeneration of blood vessels as well as the improvement of the cardiac function.
Already, the iPS cells have been taken, and then stored, from the blood cells donated by healthy individuals
On Monday, the researchers stated how they chose to carry out a clinical trial in a clinical researchs stead as they had hoped to attain, as early as possible, authorization from the health ministry for clinical applications.
There are severe evaluating risks involved in the clinical trial. These may include the possibility of cancer as well as the efficacy of transplanting many million cells per patient, which may consist of tumor cells.
In Japan, this will be marked as the second clinical trial based on iPS. The first clinical trial of such kind was carried out on patients suffering from eye-linked ailments. This was done so by the Riken research institute.
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Osaka University-based team successfully conducts first transplantation of cardiac muscle cells around the globe - Medical Herald
Consumer Claims Over Peter Thomas Roth’s Alleged "False Advertising" of Skincare Products to Go to Jury – The Fashion Law
A handful of the buzzy products of Peter Thomas Roth are at the center of a strongly-worded lawsuit, one that accuses the New York-based skincare company which boasts about its richly nourishing and technologically advanced offerings of peddling pseudoscience and falsifying the effectiveness of its hyaluronic acid-soaked skin creams and rose stem cell-formulated face masks in an attempt to stand out in the fiercely competitive $135 billion-plus skincare market and cater to the rising demand for anti-aging products among consumers.
According to the complaint that Peter Thomas Roth, LLC (Roth) customers Kari Miller and Samantha Paulson filed in a California state court in December 2018, Roth is running afoul of the law by making false claims about the capabilities of [its] products, at least some of which are among its best-selling products on Sephoras website. The plaintiffs assert that even in an industry known for hype, Roths outrageous marketing practices stand out among those of their competitors, as Roths claims about their [products] are not just hype; rather, they are demonstrably false.
Specifically, Miller and Paulson state that two of Roths product lines,the influencer-endorsed Rose Stem Cell line and the Water Drench line, are at the center of their suit, as both lines have allegedly been marketed and sold in conjunction with false and deceptive representations [about their] active ingredients rose stem cells and hyaluronic acid, respectively that have enabled Roth to profit enormously while its customers are left with overpriced, ineffective skin care products.
For instance the plaintiffs assert that in connection with its Water Drench line of products Roth represents that the active ingredient, hyaluronic acid, will draw moisture from the atmosphere into the users skin, and will hold 1,000 times its weight in water for up to 72 hours. This is impossible, they claim, as hyaluronic acid is incapable of absorbing anywhere near 1,000 times its weight in water, even when it is in its anhydrous (i.e., waterless; completely dry) form.
The judge notes that Roth softened the claim with the words up to in connection with the absorption power of thehyaluronic acid, but he also claims that subtle qualifications do not overcome the thrust of the ad, which is thatthe ad was one thousand times its weight in water.
As for Roths line of Rose Stem Cell products, which the brand claims are are capable of repairing, regenerating, and rejuvenating human skin andstimulating cellular turnoveras a result of the inclusion of rose stem cells, the plaintiffs argue that there is absolutely no evidence thatrosestem cells can provide such benefits. They allege thatRothis clearly attempting to capitalize on the recent media attention that has been given to medical research of human stem cells, with the goal of confusing consumers and causing them to erroneously believe that they will receive significant health benefits by using the Rose Stem Cell Products.
Such pseudo-science has enabled Roth to sell over-priced products to a growing market for skin care products, whileenjoying an unlawful advantage over [its] competitors, the plaintiffs assert in the suit, which has since been transferred from California state court to federal court.
In a couple of recent developments in the case, Judge William Alsup of the U.S. District Court for the Northern District of California denied the plaintiffs bid for class action approval, a move that would enable other individuals who have purchased the allegedly misrepresented products to join in their suit and any ultimate settlement sum. According to Judge Alsups January 22 decision, The plaintiffs can obtain their requested liability determination [for their false advertising claims] and statewide injunction against Roths challenged ads without certifying a class.
Meanwhile, in a separate January 22 order, the judge decided on Roths motion for summary judgment, refusing (for the most part) to issue a final decision resolving the plaintiffs claims ahead of trial because there are still issues of fact to be determined, namely whether Roths marketing claims are deceptive.
According to Judge Alsup, it is unclear how a reasonable consumer might view the marketing claims that Roth uses in connection with its Rose Stem Cell Mask namely, the labels, rose stem cells, cutting edge bio-technology, bio-repair, regenerates, and rejuvenates. While some reasonable consumers might interpret this [language] as mere puffery, and thus, not objective, actionable statements, others could sensibly conclude that rose stem cells actually repair human skin, which the plaintiffs argue is untrue, thereby, making the marketing claims deceptive.
In terms of Roths Water Drench line of products, the judge states that the plaintiffs contend the reasonable consumer would believe that hyaluronic acidactually canattract and retain one thousand times its weight in water, and in fact, a jury could find that, based on the ad, reasonable consumers would expect that hyaluronic acid absorbs and retains about one thousand times its weight in water.As such, these issues must go before a jury, which, Judge Alsup says will look forward to an in-court demonstration in which a certain amount of hyaluronic acid is placed in a beaker, one thousand times that weight in water is placed in another beaker, and the contents are combined, all watching to see if all the water will be absorbed.
*The case is Kari Miller, et al., v. Peter Thomas Roth, LLC, et al.,3:19-cv-00698 (N.D.Cal.)
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Consumer Claims Over Peter Thomas Roth's Alleged "False Advertising" of Skincare Products to Go to Jury - The Fashion Law
Win an Image Renewal Ritual Collection worth 140 from Allure – image.ie
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There are a number of peels on offer using Image products, like the Ormedic Lift treatment that promises to recharge the youthfulness factor within the skin and increase internal hydration, or the Acne Lift, which is abeta and alpha-hydroxy acid cocktail giving antibacterial, anti-inflammatory and antiseptic benefits to fragile compromised and reactive skin.
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Win an Image Renewal Ritual Collection worth 140 from Allure - image.ie
Gladstone Scientists Funded by NIH to Dive Deep Into ApoE4’s Role in Alzheimer’s Disease – P&T Community
SAN FRANCISCO, Jan. 29, 2020 /PRNewswire/ -- The story of Alzheimer's disease is familiar and heartbreaking. As neurons degenerate and die, patients slowly lose their memories, their thinking skills, and ultimately, their ability to perform basicday-to-day tasks.
For years, clinical trials investigating potential treatments for Alzheimer's disease have come up short. That's why researchers at Gladstone Institutes are delving deeper into the question of what drives this complex disease.
Now, a team led by Senior Investigator and President EmeritusRobert Mahley, MD, PhD, has received $4.8 million from the National Institutes of Health (NIH) to study a promising culprit: apoE4, a protein associated with increased risk of Alzheimer's disease.
ApoE4 is one of the forms of apolipoprotein E, a protein that aids repair processes in neurons injured by aging, stroke, or other causes. The most common form is called apoE3, but apoE4 is not rare: it is found in one-quarter of the human population and in about two-thirds of all Alzheimer's patients, which makes it the most important genetic risk factor for the disorder.
"ApoE4 dramatically rewires cellular pathways in neurons and impairs their function," Mahley said. "Our goal is to understand how this rewiring occurs and identify potential new treatment strategies to negate the detrimental effects."
ApoE3 and apoE4 differ at only a single point in the sequence of their amino acid building blocks. But that single change gives apoE4 a very different shape from apoE3, making it more susceptible to being broken down into smaller fragments within a neuron.
"Our work suggests that these apoE4 fragments are toxic to neurons and cause sweeping changes to the collection of proteins expressed within a neuron," Mahley said. "We suspect that their toxicity may underlie much of the neurodegeneration seen in Alzheimer's disease."
A Powerful Partnership
With the new NIH funding, Mahley hopes to illuminate the specifics of apoE4's toxicity in unprecedented molecular detail. Key to this work is his new partnership with Senior InvestigatorNevan Krogan, PhD, and Gladstone Mass Spectrometry Facility Director Danielle Swaney, PhD, who together have extensive expertise in studying how proteins interact with each other.
To get to the bottom of apoE4's impact, they will use a technique called affinity purification mass spectrometry (AP-MS)to first determine which proteins, out of the thousands found in a single cell, interact directly with apoE4 fragments.
"AP-MS is an important first step because it will allow us to define physical interactions between proteins that may underlie the functional deficits observed in neurons that express apoE4," Swaney said. The AP-MS work will be performed in mouse-derived neuronal cells that are similar to human neurons.
In addition to AP-MS, the collaborators will use other advanced protein analysis techniques perfected in Krogan's lab to better understand the cellular processes that are dysregulated in apoE4-expressing neurons. This additional protein work will be performed in neurons derived from human induced pluripotent stem (hiPS) cells. These stem cells are produced from human skin cells, using the procedure developed byShinya Yamanaka, MD, PhD, a Gladstone senior investigator and 2012 Nobel prize winner.
"We are quite excited to be involved in this project," Krogan said. "My lab has successfully applied AP-MS and other cutting-edge proteomic and genetic techniques to many different diseases, and we now hope to enable a much deeper understanding of apoE4."
When combined, results from the APMS work and the additional protein analyses will reveal a list of key proteins involved in processes that are specifically altered in apoE4 neurons compared to apoE3 neurons.
From that list, Mahley and Swaney will select top candidates for further investigation in neurons grown from hiPS cells. Senior InvestigatorYadong Huang, MD, PhD, who has also studied apoE4 extensively, will provide guidance on the use of the hiPS cells.
Using a gene-editing tool called CRISPR, the researchers will see if they can reverse the detrimental effects of apoE4 by activating or inhibiting genes that control their top candidate proteins in the hiPS cell-derived neurons. Finally, they will validate the findings in mice.
"By the end of the project, we hope to narrow down our list to just a few target genes or proteins that protect or restore neuronal health when we activate or inhibit them in live mice with the apoE4 gene," Swaney said. "They could then be explored as potential targets for Alzheimer's treatment in humans."
New Hope for Alzheimer's Disease
Mahley and Swaney already have some ideas about where this work may lead. Earlier this year,they publishedevidence that apoE4 broadly impacts the mitochondriaorganelles that produce the energy that powers a celland perturbs normal energy production.
"Anything could be a target at this point, but I'm particularly interested in the possibility of small-molecule drugs that could protect mitochondria from toxic apoE4 fragments," Mahley said.
Still, mitochondria are just one aspect of the bigger picture. Mahley suspects that what we call "Alzheimer's disease" is actually a collection of related conditions with different underlying causes for different patients.
"Ultimately, I think the treatment of Alzheimer's disease will be similar to the treatment of high blood pressure, in that two, three, sometimes four drugs are needed to control the disorder," he said. "So, we may need a mitochondrial protector, we may need a drug that will correctapoE4's shapeso that it is more like apoE3, and more."
Understanding the complex effects of apoE4as well as the other Alzheimer's disease-associated factorsbeing explored at Gladstonecould one day enable just such a comprehensive approach.
Media Contact:Megan McDevittmegan.mcdevitt@gladstone.ucsf.edu415.734.2019
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team-of-researchers-who-received.jpg Team of Researchers who Received the Grant Gladstone Senior Investigator and President Emeritus Bob Mahley (center) will collaborate with the director of the Gladstone Mass Spectrometry Facility, Danielle Swaney (left), and Senior Investigator Nevan Krogan (right) to uncover the mechanisms of apoE4 toxicity in Alzheimer's disease.
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Robots don’t have to be so embarrassing – The Outline
Robots are pathetic. You need only watch a robot soccer fail compilation to see that humans ancient quest to build synthetic replicas of ourselves out of nuts, bolts and wiring has been a bust. Every new, groundbreaking robot inevitably turns out to be an ungodly abomination, either physically inept or utterly incapable of social interaction. Our latest attempt at a full-on humanoid, Sophia, looks like a pre-loved department store mannequin and sounds like a 2007-era chatbot dialed to the VERY DEPRESSED setting. Shed be a walking repudiation of brainless techno-optimism, if she could actually walk.
Even attempts to build simpler, dog-like droids, such as Boston Dynamics Spot, have produced robots barely worthy of the name. They dont look much better than what youd expect from an adult Erector set enthusiasts weekend garage projects. Some people find these things terrifying, but I take my cues from the manufacturers, who seem incredibly proud when one of their creations performs a task as easy as opening a door.
Imitating human intelligence in software has also proven a task more difficult than expected. Despite the well-financed wet dreams of companies like Uber, the automotive industry has begun to quietly admit that truly self-driving cars are going to happen in decades, not just a few years from now. The Blue Brain project, which received a billion euros from the EU in 2013 and promised to simulate a human brain by 2019, did not succeed. Blue Brain seems to have had some success building a 3D atlas of a mouse brain, but the projects supercomputer, which takes up an entire room, is heaving and groaning under the strain of doing the same for a human mind. Valiant efforts to simulate a transparent, one millimetre nematode called C. elegans, ongoing since 2004, have yielded similarly slow progress. C. elegans has 302 neurons. The human brain has 86 billion.
These stuff-ups are endlessly amusing to me. I dont want to mock the engineers who pour thousands of hours into building novelty dogs made of bits of broken toasters, or even the vertiginously arrogant scientists who thought they could simulate the human brain inside a decade. (Inside a decade! I mean, my god!) Well, okay, maybe I do want to mock them. Is it a crime to enjoy watching our cultures systematic over-investment in digital Whiggery get written down in value time and time again?
On the other hand, maybe the people doing this stuff have just figured out that attaching the terms robot or artificial intelligence to whatever youre up to is a great way of attracting investment from rich idiots. Sometimes I feel naive for thinking anyone takes these wild claims seriously, but that is precisely the power of a good ideology. The promises of robotics and AI are so seductive that people suspend their critical faculties. Whether you are a business like Uber striving to eliminate the messy and expensive production input known as human beings, or a normal person desperate for easy transportation or someone to keep your elderly relatives company, the way we talk about robots and AI suggests these smart solutions are just around the corner. Even people with their heads screwed on properly dont seem to understand how credulously the media hypes up their coverage of AI.
What these doomed overreaches represent is a failure to grasp the limits of human knowledge. We dont have a comprehensive idea of how the brain works. There is no solid agreement on what consciousness really is. Is it divine? Is it matter? Can you smoke it? Do these questions even make sense? We dont know the purpose of sleep. We dont know what dreams are for. Sexual dimorphism in the brain remains a mystery. Are you picking up a pattern here? Even the seemingly quotidian mechanical abilities of the human body running, standing, gripping, and so on are not understood with the scientific precision that you might expect. How can you make a convincing replica of something if you dont even know what it is to begin with? We are cosmic toddlers waddling around in daddys shoes, pretending to work at the office by scribbling on the walls in crayon, and then wondering where our paychecks are.
The world is an astonishing place, and the idea that we have in our possession the basic tools needed to understand it is no more credible now than it was in Aristotles day, writes philosopher Thomas Nagel. But accepting this epistemic knuckle sandwich doesnt mean abandoning the pursuit of robotics.
Enter the frogbot, a living machine synthesized by a research team at the Allen Discovery Center at Tufts University in Boston.
Frogbots (called xenobots by their creators, a stupid name I refuse to use), are tiny little artificial animals made out of stem cells from the African clawed frog. They cant do much yet move around on two stumpy legs, carry tiny objects in a pouch but to me, they are stranger and scarier than any robot weve made out of metal and plastic.
A "frogbot" developed by researchers at Tufts University.
There are three basic steps to the frogbot process. First, stem cells that will develop into frog skin and frog heart are grown in a dish. (The proto-heart cells produce rhythmic contractions, which is how the finished frogbots move around.) Second, a computer runs an algorithm that simulates thousands and thousands of different frogbot designs in a virtual environment to see which ones are capable of whatever action you want them to perform. Finally, the designs that are likely to work are physically produced from clusters of stem cells using microsurgery, then let loose in another dish to see what they actually do. So far, they do pretty much whatever we want them to do, within reason.
This is very cool. Even though frogbots are tiny and stupid at the moment, they impress me way more than the conga line of faildroids weve managed to cobble together so far. Of course it makes sense to use materials from existing animals; weve been doing this using selective breeding techniques since the dawn of time. What are pigs or cows or sheep but frogbots built over thousands of years? The key innovation here is modelling selective evolution quickly, instead of standing around like idiots for millenia, waiting for hundreds of generations of dogs to fuck.
It makes perfect sense. Why try to reinvent the wheel when you could simply hijack biological processes that already exist? This is a classically human way of solving a problem, cleverer and yet also lazier than the futile pursuit of purely artificial robotics. A big congratulations to the scientists who figured this out, using only keen wit, a positive attitude, and a gigantic pile of money from the U.S. military research agency.
Yes, naturally this exciting new field of science is being used to develop weapons of war. This, not simply the prospect of new intelligences, is the upsetting thing about groundbreaking developments in robotics and AI. Will frogbots be a military invention that simply slides into everyday life, like the internet, canned food, and microwaves? Or will they be used to administer dangerous MKULTRA hallucinogens to innocent populations America decides are in its way? In a world controlled by a small and powerful elite that can essentially do whatever it wants, were forced to be suspicious of new technologies. Will the frogbot become bigger, smarter, and stronger? Yes, probably. Will it be my comrade? Thats another question entirely.
Eleanor Robertson is a writer and editor from Sydney, Australia.
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Robots don't have to be so embarrassing - The Outline
Divorce as Seen Through the Eyes of a Child – SWAAY
I have often heard the saying, "You were probably too young to remember this, but . . ." I can honestly say that I can recall quite a bit from my childhood even though I can't seem to recall what I had for breakfast yesterday. I remember a lot, including some things that I wish were fuzzy.
I know this sounds strange, but I remember my dad leaving. I was barely two years old, so obviously I was at an age when I could not fully comprehend what I was experiencing at the time, but I already knew I missed my dad and I wanted him to come home. Divorce is a topic I am very familiar with, both personally and professionally. There are countless people who seek counseling in various areas of their life and to me; that is just another day at the office. However, my story hits a different type of nerve for me. It is a story that I had processed in my own therapy, but this is the first time I am sharing it with the public, so (deep breath) . . . here we go.
As I mentioned before, my dad left when I was about eighteen months old. Just as I was trying to adapt to these changes as best as a toddler could, I met my dad's new "friend" and her kids. I remember she took my hand and walked me around where she worked. I am sure a lot was going on behind the scenes between my parents, but again I was too young to put things together at the time. Fast forward to age four or five, I was introduced to a new friend: anger! Oh, and nightmares. Plenty of them. One recurring nightmare was my dad leaving me. I would wake up screaming and crying, filled with a mixture of sadness, anger, shame, and guilt. My mom would come running into my room to comfort me as I sobbed against her shoulder.
Looking back now, I realized that the word that truly defined what I was feeling was powerless. My mom decided that she needed to do everything in her power to help me. So, she went to the bookstore and found several books that were supposed to help kids deal with their parents' divorce. She would read them to me, but they often told stories of children that I could not relate to, or they were often telling me how I should feel, rather than allowing me the space to access my own feelings. It was frustrating and overwhelming.
It is fascinating how quickly we can adapt. I started to get used to going back and forth between my two homes. However, it was only for a short period of time that I felt "okay." Fast forward again to around age ten. Just as I was starting to accept all the changes including separate homes, blended families, and different sets of rules, I had to endure a long and terrifying custody battle. I felt like my parents were playing tug of war with me in the middle! The anger that I thought had disappeared came back in full force and even brought additional feelings, including shame, grief, sadness, low self-esteem, people-pleasing tendencies, just to name a few. That voice I was working so hard on developing was silenced as I decided to just say or do what I thought would please my parents as well as others. I not only lost my voice, but I lost myself.
I also learned some interesting techniques from my mom. She created "games" for us to play including what we called "give me the bad stuff," which is where I would think of all the different things that were bothering me, shout, "I don't like this," while bundling them up into an invisible ball, and then handing them to my mom who would then pretend to throw them out the door or window.
My mom would tell me that I am just a kid, so I did not need to hold on to all this "yucky stuff" inside. It was the first time in a while that I felt like I had a voice. It was wonderful! I would also scream into or hit my pillow as if it were a punching bag. Pretty creative stuff, right? As my mom always says, "It takes a village," and boy was she right! I lucked out by having such an amazing support system at my elementary school.
My guidance counselor established a support group for children of divorced or divorcing parents, and it truly helped to normalize what I was feeling. I was able to speak to peers my own age going through the same things, which was helpful as many of my close friends could not relate to what I was experiencing. I was given safe, nonjudgmental outlets to express myself, and little by little I felt better.
So why am I sharing my story? Well, today as a therapist, I listen to other children's stories. Divorce is definitely not pretty, but it does not have to be so ugly! Whether the parents decide to "stay together for the children" or go their separate ways, children are getting pulled into the chaos. Sometimes, children will pretend they don't know what is going on or act as if they don't care, but trust me when I say it all leaves an impact.
My book, My Parents Are Getting a Divorce . . . I Wonder What Will Happen to Me, is an interactive workbook that was created by my mother and me during the terrifying custody battle that took place between my parents. I felt it was imperative that I assist as many children as possible to help them explore and uncover their innermost thoughts and feelings regarding their parents' divorce. Within the pages of the book, children are encouraged to write and draw as well as ask questions to get in touch with what is inside that needs to be healed.
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Divorce as Seen Through the Eyes of a Child - SWAAY
Stem Cell Therapy Market Predicted to Accelerate the Growth by 2017-2025 – Jewish Life News
Stem Cell Therapy Market: Snapshot
Of late, there has been an increasing awareness regarding the therapeutic potential of stem cells for management of diseases which is boosting the growth of the stem cell therapy market. The development of advanced genome based cell analysis techniques, identification of new stem cell lines, increasing investments in research and development as well as infrastructure development for the processing and banking of stem cell are encouraging the growth of the global stem cell therapy market.
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One of the key factors boosting the growth of this market is the limitations of traditional organ transplantation such as the risk of infection, rejection, and immunosuppression risk. Another drawback of conventional organ transplantation is that doctors have to depend on organ donors completely. All these issues can be eliminated, by the application of stem cell therapy. Another factor which is helping the growth in this market is the growing pipeline and development of drugs for emerging applications. Increased research studies aiming to widen the scope of stem cell will also fuel the growth of the market. Scientists are constantly engaged in trying to find out novel methods for creating human stem cells in response to the growing demand for stem cell production to be used for disease management.
It is estimated that the dermatology application will contribute significantly the growth of the global stem cell therapy market. This is because stem cell therapy can help decrease the after effects of general treatments for burns such as infections, scars, and adhesion. The increasing number of patients suffering from diabetes and growing cases of trauma surgery will fuel the adoption of stem cell therapy in the dermatology segment.
Global Stem Cell Therapy Market: Overview
Also called regenerative medicine, stem cell therapy encourages the reparative response of damaged, diseased, or dysfunctional tissue via the use of stem cells and their derivatives. Replacing the practice of organ transplantations, stem cell therapies have eliminated the dependence on availability of donors. Bone marrow transplant is perhaps the most commonly employed stem cell therapy.
Osteoarthritis, cerebral palsy, heart failure, multiple sclerosis and even hearing loss could be treated using stem cell therapies. Doctors have successfully performed stem cell transplants that significantly aid patients fight cancers such as leukemia and other blood-related diseases.
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Global Stem Cell Therapy Market: Key Trends
The key factors influencing the growth of the global stem cell therapy market are increasing funds in the development of new stem lines, the advent of advanced genomic procedures used in stem cell analysis, and greater emphasis on human embryonic stem cells. As the traditional organ transplantations are associated with limitations such as infection, rejection, and immunosuppression along with high reliance on organ donors, the demand for stem cell therapy is likely to soar. The growing deployment of stem cells in the treatment of wounds and damaged skin, scarring, and grafts is another prominent catalyst of the market.
On the contrary, inadequate infrastructural facilities coupled with ethical issues related to embryonic stem cells might impede the growth of the market. However, the ongoing research for the manipulation of stem cells from cord blood cells, bone marrow, and skin for the treatment of ailments including cardiovascular and diabetes will open up new doors for the advancement of the market.
Global Stem Cell Therapy Market: Market Potential
A number of new studies, research projects, and development of novel therapies have come forth in the global market for stem cell therapy. Several of these treatments are in the pipeline, while many others have received approvals by regulatory bodies.
In March 2017, Belgian biotech company TiGenix announced that its cardiac stem cell therapy, AlloCSC-01 has successfully reached its phase I/II with positive results. Subsequently, it has been approved by the U.S. FDA. If this therapy is well- received by the market, nearly 1.9 million AMI patients could be treated through this stem cell therapy.
Another significant development is the granting of a patent to Israel-based Kadimastem Ltd. for its novel stem-cell based technology to be used in the treatment of multiple sclerosis (MS) and other similar conditions of the nervous system. The companys technology used for producing supporting cells in the central nervous system, taken from human stem cells such as myelin-producing cells is also covered in the patent.
Global Stem Cell Therapy Market: Regional Outlook
The global market for stem cell therapy can be segmented into Asia Pacific, North America, Latin America, Europe, and the Middle East and Africa. North America emerged as the leading regional market, triggered by the rising incidence of chronic health conditions and government support. Europe also displays significant growth potential, as the benefits of this therapy are increasingly acknowledged.
Asia Pacific is slated for maximum growth, thanks to the massive patient pool, bulk of investments in stem cell therapy projects, and the increasing recognition of growth opportunities in countries such as China, Japan, and India by the leading market players.
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Global Stem Cell Therapy Market: Competitive Analysis
Several firms are adopting strategies such as mergers and acquisitions, collaborations, and partnerships, apart from product development with a view to attain a strong foothold in the global market for stem cell therapy.
Some of the major companies operating in the global market for stem cell therapy are RTI Surgical, Inc., MEDIPOST Co., Ltd., Osiris Therapeutics, Inc., NuVasive, Inc., Pharmicell Co., Ltd., Anterogen Co., Ltd., JCR Pharmaceuticals Co., Ltd., and Holostem Terapie Avanzate S.r.l.
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Stem Cell Therapy Market Predicted to Accelerate the Growth by 2017-2025 - Jewish Life News
Rapid analysis shows that the 2019-nCoV coronavirus resembles viruses from bats – Massive Science
The 2019 novel coronavirus (2019-nCoV) outbreak has sparked a speedy response, with scientists, physicians, and front-line healthcare professionals analyzing data in real-time in order to share findings and call out misinformation. Today, The Lancet published two new peer-reviewed studies: one which found that the new coronavirus is genetically distinct from human SARS and MERS, related viruses which caused their own outbreaks, and a second which reports clinical observations of 99 individuals with 2019-nCoV.
The first cases of the coronavirus outbreak were reported in late December 2019. In this new study, Nanshan Chen and colleagues analyzed available clinical, demographic, and laboratory data for 99 confirmed coronavirus cases at the Wuhan Jinyintan Hospital between Jan 1 to Jan 20, 2020, with clinical outcomes followed until 25th January.
Chen and colleagues reported that the average age of the 99 individuals with 2019-nCoV is around 55.5 years, where 51 have additional chronic conditions, including cardiovascular and cerebrovascular (blood flow to the brain) diseases. Clinical features of the 2019-nCoV include a fever, cough, shortness of breath, headaches, and a sore throat. 17 individuals went on to develop acute respiratory distress syndrome, resulting in death by multiple organ failure in 11 individuals. However, it is important to note here that most of the 2019-nCoV cases were treated with antivirals (75 individuals), antibiotics (70) and oxygen therapy (75), with promising prognoses, where 31 individuals were discharged as of 25th January.
Based on this sample, the study suggests that the 2019 coronavirus is more likely to affect older men already living with chronic conditions but as this study only includes 99 individuals with confirmed cases, it may not present a complete picture of the outbreak. As of right now, there are over 6,000 confirmed coronavirus cases reported, where a total of 126 individuals have recovered, and 133 have died.
In a second Lancet study, Roujian Lu and their fellow colleagues carried out DNA sequencing on samples, obtained from either a throat swab or bronchoalveolar lavage fluids, from eight individuals who had visited the Huanan seafood market in Wuhan, China, and one individual who stayed in a hotel near the market. Upon sequencing the coronaviruss genome, the researchers carried out phylogenetic analysis to narrow down the viruss likely evolutionary origin, and homology modelling to explore the virus receptor-binding properties.
Lu and their fellow colleagues found that the 2019-nCoV genome sequences obtained from the nine patients were very similar (>99.98% similarity). Upon comparing the genome to other coronaviruses (like SARS), the researchers found that the 2019-nCoV is more closely related (~87% similarity) to two bat-derived SARS-like coronaviruses, but does not have as high genetic similarity to known human-infecting coronaviruses, including the SARS-CoV (~79%) orMiddle Eastern Respiratory Syndrome (MERS) CoV (~50%).
The study also found that the 2019-nCoV has a similar receptor-binding structure like that of SARS-CoV, though there are small differences in certain areas. This suggests that like the SARS-CoV, the 2019-nCoV may use the same receptor (called ACE2) to enter cells, though confirmation is still needed.
Finally, phylogenetic analysis found that the 2019-nCoV belongs to the Betacoronavirus family the same category that bat-derived coronaviruses fall into suggesting that bats may indeed be the 2019-nCoV reservoir. However, the researchers note that most bat species are hibernating in late December, and that no bats were being sold at the Huanan seafood market, suggesting that while bats may be the initial host, there may have been a secondary animal species which transmitted the 2019-nCoV between bats and humans.
Its clear that we can expect new findings from the research community in the coming days as scientists attempt to narrow down the source of the 2019-nCoV.
Read this article:
Rapid analysis shows that the 2019-nCoV coronavirus resembles viruses from bats - Massive Science
My cat’s coat is mostly white with dark tabby patches. What’s going on? – Massive Science
The 2019 novel coronavirus (2019-nCoV) outbreak has sparked a speedy response, with scientists, physicians, and front-line healthcare professionals analyzing data in real-time in order to share findings and call out misinformation. Today, The Lancet published two new peer-reviewed studies: one which found that the new coronavirus is genetically distinct from human SARS and MERS, related viruses which caused their own outbreaks, and a second which reports clinical observations of 99 individuals with 2019-nCoV.
The first cases of the coronavirus outbreak were reported in late December 2019. In this new study, Nanshan Chen and colleagues analyzed available clinical, demographic, and laboratory data for 99 confirmed coronavirus cases at the Wuhan Jinyintan Hospital between Jan 1 to Jan 20, 2020, with clinical outcomes followed until 25th January.
Chen and colleagues reported that the average age of the 99 individuals with 2019-nCoV is around 55.5 years, where 51 have additional chronic conditions, including cardiovascular and cerebrovascular (blood flow to the brain) diseases. Clinical features of the 2019-nCoV include a fever, cough, shortness of breath, headaches, and a sore throat. 17 individuals went on to develop acute respiratory distress syndrome, resulting in death by multiple organ failure in 11 individuals. However, it is important to note here that most of the 2019-nCoV cases were treated with antivirals (75 individuals), antibiotics (70) and oxygen therapy (75), with promising prognoses, where 31 individuals were discharged as of 25th January.
Based on this sample, the study suggests that the 2019 coronavirus is more likely to affect older men already living with chronic conditions but as this study only includes 99 individuals with confirmed cases, it may not present a complete picture of the outbreak. As of right now, there are over 6,000 confirmed coronavirus cases reported, where a total of 126 individuals have recovered, and 133 have died.
In a second Lancet study, Roujian Lu and their fellow colleagues carried out DNA sequencing on samples, obtained from either a throat swab or bronchoalveolar lavage fluids, from eight individuals who had visited the Huanan seafood market in Wuhan, China, and one individual who stayed in a hotel near the market. Upon sequencing the coronaviruss genome, the researchers carried out phylogenetic analysis to narrow down the viruss likely evolutionary origin, and homology modelling to explore the virus receptor-binding properties.
Lu and their fellow colleagues found that the 2019-nCoV genome sequences obtained from the nine patients were very similar (>99.98% similarity). Upon comparing the genome to other coronaviruses (like SARS), the researchers found that the 2019-nCoV is more closely related (~87% similarity) to two bat-derived SARS-like coronaviruses, but does not have as high genetic similarity to known human-infecting coronaviruses, including the SARS-CoV (~79%) orMiddle Eastern Respiratory Syndrome (MERS) CoV (~50%).
The study also found that the 2019-nCoV has a similar receptor-binding structure like that of SARS-CoV, though there are small differences in certain areas. This suggests that like the SARS-CoV, the 2019-nCoV may use the same receptor (called ACE2) to enter cells, though confirmation is still needed.
Finally, phylogenetic analysis found that the 2019-nCoV belongs to the Betacoronavirus family the same category that bat-derived coronaviruses fall into suggesting that bats may indeed be the 2019-nCoV reservoir. However, the researchers note that most bat species are hibernating in late December, and that no bats were being sold at the Huanan seafood market, suggesting that while bats may be the initial host, there may have been a secondary animal species which transmitted the 2019-nCoV between bats and humans.
Its clear that we can expect new findings from the research community in the coming days as scientists attempt to narrow down the source of the 2019-nCoV.
Read more from the original source:
My cat's coat is mostly white with dark tabby patches. What's going on? - Massive Science
UAB: 50 years of Improving Birmingham, Alabama and the World – Birmingham Times
UAB Magazine
Written by Charles Buchanan, Brett Bralley and Jay Taylor with editorial contributions from Matt Windsor and UAB Public Relations. Images from UAB Archives, Rachel Hendrix, Andrea Mabry, Sarah Parcak, Steve Wood and Getty Images. Web design by Tyler Bryant. Reprinted by permission of UAB Magazine.
UABs birth was like a ray of sunlight punching through the smog.
In 1969 the newly independent university, uniting a pioneering academic medical center and a growing extension center, brought the promise of a brighter future to a city eager for change.
Birmingham is better because of UAB. So are Alabama, America, and the world. In the following pages, discover some of the many ways that UAB has fulfilled its promiseby saving lives, solving problems, expanding knowledge, and opening doorsover 50 years.
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Best of the best
UABs accolades shine a global spotlight on Birmingham and Alabama:
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A way to retrain the brain
Most scientists once believed that neuroplasticitythe brains ability to grow or repair itselfended in childhood. But research by UAB neuroscientist Edward Taub, Ph.D., contributed to a shift in thinking, and in the 1990s he developed constraint-induced (CI) therapy for stroke patients with poorly functioning limbs. As the intensive training helps patients learn to accomplish tasks with their affected limbs, the brain adapts by strengthening communication with those parts of the body. And the results have been remarkable: Most patients see a clinically significant level of improvement in their ability to use their affected limbs, and brain scans have shown an increase in gray matter. Taub and UAB clinical psychologist Gitendra Uswatte, Ph.D., have used CI therapy to help thousands of stroke patientsand adapted it for patients impacted by cerebral palsy, traumatic brain injury, multiple sclerosis, and spinal cord injury. Today CI therapy is in use worldwide.
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Discoveries on ice
UAB scientists conduct a lot of research in the fieldbut none may go as far afield as James McClintock, Ph.D.; Charles Amsler, Ph.D.; and Maggie Amsler. Their investigations take place at Palmer Station, Antarctica6,898 miles from their campus offices. For two decades, the biologists have led teams that dive into the frigid waters surrounding the icy continent to study the chemical ecology of the unique marine algae and invertebrates living there. What theyve discovered could aid the search for new drugs to help humans. The group also chronicles the dramatic impact of climate change, such as ocean acidification, on Antarctic marine life. You can see climate change happening there like no other place on earth, says McClintock.
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A pinch of prevention
UAB endocrinologist Constance Pittman, M.D., turned her research passioniodines impact on thyroid functioninto a global mission. In the 1990s and 2000s, she teamed up with Kiwanis International and UNICEF to help eradicate iodine deficiency disorders (IDD), a prevalent cause of cognitive disabilities. Pittman traveled the world to convince companies to add iodine to table saltthe simplest solution for preventing IDD. And her work helped make a lasting impact.
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Target: Diabetes
In 1973, UAB opened the nations first public diabetes hospitaland the first linked with an academic medical center. Today physicians on the front lines of the diabetes epidemic have an exciting new option to help their patients, thanks to breakthrough research from UABs Comprehensive Diabetes Center.
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Sharing stories that matter
WBHM 90.3 FM radio went on the air in 1976 as the 200th National Public Radio (NPR)-affiliated station. A member-supported service of UAB, WBHM provides global news and award-winning local coverage to Birmingham and the surrounding region. The station also recently welcomed StoryCorps, an NPR-affiliated initiative, to collect stories from the Birmingham community that will be housed at the Library of Congress in Washington, D.C.
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Book of Life
Its tough to find a physician anywhere in the world who hasnt learned a few things from Tinsley Harrison, M.D. The legendary School of Medicine cardiologist and dean created and edited Harrisons Principles of Internal Medicine, which has been reprinted 20 times, translated into 14 languages, and become arguably the most recognized book in all of medicine, according to the Journal of the American Medical Association.
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Foresight
The School of Optometry has been a pioneer since it opened in 1969 as the nations first optometry school associated with an academic medical center. Three years later, it became the first optometry teaching program affiliated with a Veterans Administration (VA) hospital, establishing a national model. Today more than 2,500 optometry staff and students from various schools work in the VA system nationwide.
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Helping our hometown
Living and working in the heart of the city, UAB students, faculty, and staff cant help but feel a connection to Birmingham. Here are just a few ways Blazers have volunteered to support their neighbors:
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A whole new ball game
Gene Bartow Mens basketball coach1977-1996
UAB started a winning tradition in 1977 when it hired coach Gene Bartow away from powerhouse UCLA to start a mens basketball program. He created a legendary team able to topple top rivals and reach the NCAA Tournament in just its third seasonthe first of 15 NCAA Tournament and 12 National Invitational Tournament appearances on its record. As UABs first athletic director, Bartow also helped UAB compete in other arenas. Today student-athletes in 18 sports give Birmingham reasons to cheer. Take a spin through some of the Blazers most memorable moments:
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New views of history
Its as if Indiana Jones and Google Earth had a love child. Thats how UAB anthropology faculty member and National Geographic fellow Sarah Parcak, Ph.D., described space archaeology to Stephen Colbert on The Late Show in 2016. She has pioneered the use of high-resolution satellite imagery to search for the buried remains of lost civilizations. And her discoveries have thrilled people worldwide, including Colbert. She was even mentioned in a Jeopardy! answer earlier this year.
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Defense team
UAB immunologists have been among the first to shed light on the mechanisms powering our bodys defenses:
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Game changers
Future football helmets may better protect athletes thanks to mechanical engineering professor Dean Sicking, Ph.D. (Before coming to UAB, he developed the lifesaving SAFER barriers used on NASCAR and IndyCar courses.) Analyzing data from thousands of helmet-to-helmet impacts in football, Sicking has developed designs for a new helmet that could address concussionsabsorbing as much energy of the impact as possible so that the athlete has less risk of brain injury.
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The dividends of discovery
In 2018-2019, UAB received $602 million in research grants and awardsjust one year after surpassing the $500-million milestone for the first time. We are aiming high and exceeding our goals, and it is a testament to the UAB research communitys great ideas, hard work, and will to succeed, says Christopher Brown, Ph.D., vice president for research. A rise in research funding means more opportunities to explore the frontiers of knowledgebut it also enables UAB to attract top minds from around the country in health care, engineering, the sciences, and more, plus create new jobs that boost the local economy. Want to ensure that UAB continues its upward trajectory? Philanthropic support helps position the university to attain competitive research grants.
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Giant leaps
Space is the place for UAB people and technology:
Researcher Larry DeLucas, O.D., Ph.D., became the first optometrist in orbit with a 1992 mission aboard the shuttle Columbia. There he conducted experiments to grow protein crystals, which give scientists a 3D view of protein structuresand a greater understanding of the roles they play in disease. DeLucas also served as chief scientist for the International Space Station in 1994-1995.
Astrophysicist Thomas Wdowiak, Ph.D., passed away in 2013, but his name lives onon Mars. The Red Planets Wdowiak Ridge honors the physics faculty members role in NASAs Mars Exploration Project. Wdowiak was in charge of operating the Mossbauer spectrometers onboard the Spirit and Opportunity rovers that helped uncover firm evidence that water once existed on Mars.
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Focus on finances
Would you like to get better at saving, budgeting, or investing? Or do you dream of launching a business? The Regions Institute for Financial Education in the Collat School of Business has been helping people throughout the community develop practical, lifelong financial management skills since 2015. Some of its programs include a Money Math Camp for middle schoolers, a College Bridge Camp to prepare high schoolers for life after graduation, and for adults, a Do-It-Yourself Credit Repair Workshop.
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Going green
Campus expansions have reshaped Birminghams Southside, and UAB works hard to be a good steward of that spaceand set a sustainable example. In 2008, UAB brought open green space into the heart of Birmingham by converting a city street into the Campus Green. Now UAB is aiming to reduce its greenhouse gas emissions by 20 percent and establish a clean energy standard of 20-percent renewable energy by 2025.
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Ingenuity vs. Infection
Virus vanguards
Antiviral therapies are essential for treating everything from influenza to HIV. In 1977, UAB pediatrics experts Richard Whitley, M.D., and Charles Alford, M.D., helped spark the antiviral revolution by developing vidarabine, the first drug to treat encephalitis caused by the herpes simplex virus. In the 1990s, Whitley and his team transformed the herpes virus into a genetically engineered weapon against tumors.
Vaccines for everyone
The laboratory of Moon Nahm, M.D., is a national treasure, notes the National Institutes of Health. But its discoveries could help protect millions of children worldwide threatened by S. pneumoniae infections, the leading cause of pneumonia. (Nahms lab also is designated a World Health Organization Pneumococcal Reference Laboratory.) His mission is to make pneumonia vaccines more affordable for use in developing countries.
Global guardian
GeoSentinel is a worldwide network of clinics watching for potential pandemics in an increasingly interconnected world, ready to relay information quickly about new disease outbreaks and effective treatments. And it has Alabama roots. UAB travel medicine expert David Freedman, M.D., cofounded GeoSentinel, a collaboration between the International Society for Travel Medicine and the Centers for Disease Control and Prevention, in the 1990s. He also directed the network for 20 years.
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Staying safe on the road
In 2002, UAB public health researchers unveiled the Digital Childa pioneering computer model evaluating the physical consequences of car crashes on young passengers at various stages of developmentto generate data that could lead to improved child safety devices. Shift gears to today, and researchers in UABs TRIP (Translational Research for Injury Prevention) Lab use virtual realitya first-of-its-kind SUV simulator built with Honda Manufacturing of Alabamato study distracted driving in an effort to save lives. The TRIP Lab also has a portable simulator for schools and community events to help educate students and others on the dangers of distracted driving.
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A home for Birmingham history
Odessa WoolfolkEducator and civic leader
When Birmingham first dreamed of developing a civil rights museum and research center, UABs Odessa Woolfolk, then special assistant to the president and director of community relations, and Horace Huntley, Ph.D., a historian and first director of the African American studies program, helped lead efforts to turn that idea into a reality. The Birmingham Civil Rights Institute opened in 1992, with Woolfolk as president of its board of directors. Huntley also directed the institutes Oral History Project, which preserves the accounts of foot soldiers and other witnesses to the Birmingham campaign. Today the BCRI attracts visitors from around the world and is a key component of the Birmingham Civil Rights National Monument.
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Invention in action
Faculty, staff, and students are designing the future for the rest of us. Preview some of their ingenious solutions:
Each year, biomedical engineering and business students develop technologies to help people overcome physical limitations. Examples include a joystick-controlled wheelchair for toddlerswhich won an international awardbuilt for the Bell Center for Early Intervention Programs, and a special scale to help wheelchair users monitor their weight, used by the Lakeshore Foundation. Another design, a mechanical umbrella to protect power wheelchair users from rainy weather, scored second place at the 2018 World Congress on Biomechanics.
Graphic design students in UABs Bloom Studio unleash their talents to support local nonprofits and underserved communities. You can spot their work on license plates and signs that promote and protect the Cahaba Riverpart of a collaboration with the Cahaba River Society.
Solution Studios pairs Honors College, engineering, and nursing students with UAB health professionals to tackle everyday problems affecting patient care. One team has designed a device prototype that could improve quality of life for patients wearing ostomy bags to expel waste. Another has focused on new, more comfortable methods of applying wires to the skin in settings such as intensive care units.
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Spreading the word
Low literacy levels translate into increased high school dropout rates, a lower-performing workforce, and higher rates of social problems, say UAB School of Education experts. For years UABs Maryann Manning, Ed.D., led the charge to improve literacy across Alabama, launching programs such as a conference that attracted thousands of local schoolchildren to share their writing with authors and illustrators. Today the Maryann Manning Family Literacy Center continues her legacy, providing enrichment activities in reading, writing, math, arts, and science for children and helping teachers across Alabama learn innovative strategies to foster literacy skills in their classrooms.
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The heart of innovation
John Kirklin, M.D.Surgery superstar
John Kirklin, M.D., helped put Birmingham on the medical map when he was recruited in 1966 to chair the Department of Surgery. He already was a superstar at the Mayo Clinic, where he had revolutionized cardiovascular surgery by improving the heart-lung machine and performing the first operations for a variety of congenital heart malformations. At UAB he continued to pursue new methods and techniques, such as the development of a computerized intensive care unit with continuous monitoring of vital functions, which became a model for ICUs worldwide.
When Kirklin passed away in 2004, colleagues estimated his medical innovations had saved millions of lives. And his legacy thrives in other ways: UAB is a world-class medical center in part because of Kirklins work behind the scenes, where he championed the combination of public and private investments to foster growth. His textbook, Cardiac Surgery, remains a must-read for anyone in the field. His name lives on in The Kirklin Clinic of UAB Hospital, which opened in 1992. And his son, cardiothoracic surgeon James Kirklin, M.D., directs UABs James and John Kirklin Institute for Research in Surgical Outcomes.
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Birthplace of new businesses
UABs ideas and energy are an engine for entrepreneurship. The university was a founder of Birminghams Innovation Depot, where start-up companiessome born from UAB research breakthroughsfind the resources they need to grow. Today Innovation Depot is the Southeasts largest high-tech business incubator, home to more than 100 companies.
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University of opportunity
In the fall of 2019, underrepresented students made up nearly 42 percent of UABs enrollment, and 20.5 percent of undergraduates were first-generation students. UAB has a long history of widening access to higher educationand potential careers in science and health careamong diverse students. Back in 1978, the Minority High School Research Apprentice Program began matching local students with faculty members for summer research experiences. Today, initiatives such as the Department of Surgerys Pre-College Internship for Students from Minority Backgrounds and the Neuroscience Roadmap Scholars program offer similar opportunities for students along their educational journeys.
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Successful careers begin here
More than 135,000 alumni call UAB their alma mater. Today youll find them across the United States and around the world, working as leaders in health care, science, business, art, engineering, government, education, and other fields. Many stay connected with UAB through the National Alumni Society, which was established in 1979 and has 63 chapters in locations ranging from Washington, D.C., to Taiwan.
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Follow this link:
UAB: 50 years of Improving Birmingham, Alabama and the World - Birmingham Times
What Is CRISPR? | Live Science
CRISPR technology is a simple yet powerful tool for editing genomes. It allows researchers to easily alter DNA sequences and modify gene function. Its many potential applications include correcting genetic defects, treating and preventing the spread of diseases and improving crops. However, its promise also raises ethical concerns.
In popular usage, "CRISPR" (pronounced "crisper") is shorthand for "CRISPR-Cas9." CRISPRs are specialized stretches of DNA. The protein Cas9 (or "CRISPR-associated") is an enzyme that acts like a pair of molecular scissors, capable of cutting strands of DNA.
CRISPR technology was adapted from the natural defense mechanisms of bacteria and archaea (the domain of single-celled microorganisms). These organisms use CRISPR-derived RNA and various Cas proteins, including Cas9, to foil attacks by viruses and other foreign bodies. They do so primarily by chopping up and destroying the DNA of a foreign invader. When these components are transferred into other, more complex, organisms, it allows for the manipulation of genes, or "editing."
Until 2017, no one really knew what this process looked like. In a paper published Nov. 10, 2017, in the journal Nature Communications, a team of researchers led by Mikihiro Shibata of Kanazawa University and Hiroshi Nishimasu of the University of Tokyo showed what it looks like when a CRISPR is in action for the very first time. [A Breathtaking New GIF Shows CRISPR Chewing Up DNA]
CRISPRs: "CRISPR" stands for "clusters of regularly interspaced short palindromic repeats." It is a specialized region of DNA with two distinct characteristics: the presence of nucleotide repeats and spacers. Repeated sequences of nucleotides the building blocks of DNA are distributed throughout a CRISPR region. Spacers are bits of DNA that are interspersed among these repeated sequences.
In the case of bacteria, the spacers are taken from viruses that previously attacked the organism. They serve as a bank of memories, which enables bacteria to recognize the viruses and fight off future attacks.
This was first demonstrated experimentally by Rodolphe Barrangou and a team of researchers at Danisco, a food ingredients company. In a 2007 paper published in the journal Science, the researchers used Streptococcus thermophilus bacteria, which are commonly found in yogurt and other dairy cultures, as their model. They observed that after a virus attack, new spacers were incorporated into the CRISPR region. Moreover, the DNA sequence of these spacers was identical to parts of the virus genome. They also manipulated the spacers by taking them out or putting in new viral DNA sequences. In this way, they were able to alter the bacteria's resistance to an attack by a specific virus. Thus, the researchers confirmed that CRISPRs play a role in regulating bacterial immunity.
CRISPR RNA (crRNA): Once a spacer is incorporated and the virus attacks again, a portion of the CRISPR is transcribed and processed into CRISPR RNA, or "crRNA." The nucleotide sequence of the CRISPR acts as a template to produce a complementary sequence of single-stranded RNA. Each crRNA consists of a nucleotide repeat and a spacer portion, according to a 2014 review by Jennifer Doudna and Emmanuelle Charpentier, published in the journal Science.
Cas9: The Cas9 protein is an enzyme that cuts foreign DNA.
The protein typically binds to two RNA molecules: crRNA and another called tracrRNA (or "trans-activating crRNA"). The two then guide Cas9 to the target site where it will make its cut. This expanse of DNA is complementary to a 20-nucleotide stretch of the crRNA.
Using two separate regions, or "domains" on its structure, Cas9 cuts both strands of the DNA double helix, making what is known as a "double-stranded break," according to the 2014 Science article.
There is a built-in safety mechanism, which ensures that Cas9 doesn't just cut anywhere in a genome. Short DNA sequences known as PAMs ("protospacer adjacent motifs") serve as tags and sit adjacent to the target DNA sequence. If the Cas9 complex doesn't see a PAM next to its target DNA sequence, it won't cut. This is one possible reason that Cas9 doesn't ever attack the CRISPR region in bacteria, according to a 2014 review published in Nature Biotechnology.
The genomes of various organisms encode a series of messages and instructions within their DNA sequences. Genome editing involves changing those sequences, thereby changing the messages. This can be done by inserting a cut or break in the DNA and tricking a cell's natural DNA repair mechanisms into introducing the changes one wants. CRISPR-Cas9 provides a means to do so.
In 2012, two pivotal research papers were published in the journals Science and PNAS, which helped transform bacterial CRISPR-Cas9 into a simple, programmable genome-editing tool.
The studies, conducted by separate groups, concluded that Cas9 could be directed to cut any region of DNA. This could be done by simply changing the nucleotide sequence of crRNA, which binds to a complementary DNA target. In the 2012 Science article, Martin Jinek and colleagues further simplified the system by fusing crRNA and tracrRNA to create a single "guide RNA." Thus, genome editing requires only two components: a guide RNA and the Cas9 protein.
"Operationally, you design a stretch of 20 [nucleotide] base pairs that match a gene that you want to edit," said George Church, a professor of genetics at Harvard Medical School. An RNA molecule complementary to those 20 base pairs is constructed. Church emphasized the importance of making sure that the nucleotide sequence is found only in the target gene and nowhere else in the genome. "Then the RNA plus the protein [Cas9] will cut like a pair of scissors the DNA at that site, and ideally nowhere else," he explained.
Once the DNA is cut, the cell's natural repair mechanisms kick in and work to introduce mutations or other changes to the genome. There are two ways this can happen. According to the Huntington's Outreach Project at Stanford (University), one repair method involves gluing the two cuts back together. This method, known as "non-homologous end joining," tends to introduce errors. Nucleotides are accidentally inserted or deleted, resulting in mutations, which could disrupt a gene. In the second method, the break is fixed by filling in the gap with a sequence of nucleotides. In order to do so, the cell uses a short strand of DNA as a template. Scientists can supply the DNA template of their choosing, thereby writing-in any gene they want, or correcting a mutation.
CRISPR-Cas9 has become popular in recent years. Church notes that the technology is easy to use and is about four times more efficient than the previous best genome-editing tool (called TALENS).
In 2013, the first reports of using CRISPR-Cas9 to edit human cells in an experimental setting were published by researchers from the laboratories of Church and Feng Zhang of the Broad Institute of the Massachusetts Institute of Technology and Harvard. Studies using in vitro (laboratory) and animal models of human disease have demonstrated that the technology can be effective in correcting genetic defects. Examples of such diseases include cystic fibrosis, cataracts and Fanconi anemia, according to a 2016 review article published in the journal Nature Biotechnology. These studies pave the way for therapeutic applications in humans.
"I think the public perception of CRISPR is very focused on the idea of using gene editing clinically to cure disease," said Neville Sanjana of the New York Genome Center and an assistant professor of biology, neuroscience and physiology at New York University. "This is no doubt an exciting possibility, but this is only one small piece."
CRISPR technology has also been applied in the food and agricultural industries to engineer probiotic cultures and to vaccinate industrial cultures (for yogurt, for example) against viruses. It is also being used in crops to improve yield, drought tolerance and nutritional properties.
One other potential application is to create gene drives. These are genetic systems, which increase the chances of a particular trait passing on from parent to offspring. Eventually, over the course of generations, the trait spreads through entire populations, according to the Wyss Institute. Gene drives can aid in controlling the spread of diseases such as malaria by enhancing sterility among the disease vector female Anopheles gambiae mosquitoes according to the 2016 Nature Biotechnology article. In addition, gene drives could also be used to eradicate invasive species and reverse pesticide and herbicide resistance, according to a 2014 article by Kenneth Oye and colleagues, published in the journal Science.
However, CRISPR-Cas9 is not without its drawbacks.
"I think the biggest limitation of CRISPR is it is not a hundred percent efficient," Church told Live Science. Moreover, the genome-editing efficiencies can vary. According to the 2014 Science article by Doudna and Charpentier, in a study conducted in rice, gene editing occurred in nearly 50 percent of the cells that received the Cas9-RNA complex. Whereas, other analyses have shown that depending on the target, editing efficiencies can reach as high as 80 percent or more.
There is also the phenomenon of "off-target effects," where DNA is cut at sites other than the intended target. This can lead to the introduction of unintended mutations. Furthermore, Church noted that even when the system cuts on target, there is a chance of not getting a precise edit. He called this "genome vandalism."
The many potential applications of CRISPR technology raise questions about the ethical merits and consequences of tampering with genomes.
In the 2014 Science article, Oye and colleagues point to the potential ecological impact of using gene drives. An introduced trait could spread beyond the target population to other organisms through crossbreeding. Gene drives could also reduce the genetic diversity of the target population.
Making genetic modifications to human embryos and reproductive cells such as sperm and eggs is known as germline editing. Since changes to these cells can be passed on to subsequent generations, using CRISPR technology to make germline edits has raised a number of ethical concerns.
Variable efficacy, off-target effects and imprecise edits all pose safety risks. In addition, there is much that is still unknown to the scientific community. In a 2015 article published in Science, David Baltimore and a group of scientists, ethicists and legal experts note that germline editing raises the possibility of unintended consequences for future generations "because there are limits to our knowledge of human genetics, gene-environment interactions, and the pathways of disease (including the interplay between one disease and other conditions or diseases in the same patient)."
Other ethical concerns are more nuanced. Should we make changes that could fundamentally affect future generations without having their consent? What if the use of germline editing veers from being a therapeutic tool to an enhancement tool for various human characteristics?
To address these concerns, the National Academies of Sciences, Engineering and Medicine put together a comprehensive report with guidelines and recommendations for genome editing.
Although the National Academies urge caution in pursuing germline editing, they emphasize "caution does not mean prohibition." They recommend that germline editing be done only on genes that lead to serious diseases and only when there are no other reasonable treatment alternatives. Among other criteria, they stress the need to have data on the health risks and benefits and the need for continuous oversight during clinical trials. They also recommend following up on families for multiple generations.
There have been many recent research projects based around CRISPR. "The pace of basic research discoveries has exploded, thanks to CRISPR," said biochemist and CRISPR expert Sam Sternberg, the group leader of technology development at Berkeley, California-based Caribou Biosciences Inc., which is developing CRISPR-based solutions for medicine, agriculture, and biological research.
Here are some of the most recent findings:
Additional reporting by Alina Bradford, Live Science contributor.
Additional resources
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What Is CRISPR? | Live Science
Mammoth Biosciences Raises $45 Million For Crispr DiagnosticsAnd Its Tech Is Already Being Used Against Coronavirus – Forbes
Mammoth Biosciences cofounders Janice Chen, Lucas Harrington and Trevor Martin.
Mammoth Biosciences, a company that uses gene-editing technology Crispr for disease testing, said Thursday that it had raised $45 million in Series B funding to expand into treatments. The round, led by Decheng Capital and including new investor Verily, brings total funding to over $70 million.
The South San Francisco-based company, founded in 2017 by Forbes Under 30 honorees Trevor Martin, Janice Chen, Lucas Harrington and Crispr pioneer Jennifer Doudna, uses Crispr as a genetic search engine to find disease markers and alert researchers of their presence. Theyve already partnered with others, such as gene-editing company Horizon Discovery and a UC San Francisco researcher who is creating a rapid diagnostic test to identify people infected with the new coronavirus.
The company has been one of the most prolific innovators in the overall Crispr ecosystem, says Ursheet Parikh, an investor at the Mayfield Fund, which also participated in the round.
The new capital will allow Mammoth to expand into more traditional gene editing, which can be used to treat diseases. The company also plans to double in size, Martin says. Mammoth has already moved into new lab space on the South San Francisco campus of Verily, Alphabets life sciences company.
Crispr gene editing emerged in the 2010s as a tool that could quickly and precisely snip, repair or insert genes into DNA, giving rise to companies including eGenesis, Caribou Biosciences and Sherlock Biosciences. Most biotech companies in the gene-editing space use the Crispr system with Cas9, a large protein that can cut DNA. Mammoth focuses on a different one: Cas14. Martin refers to this protein as nano-Cas, because its smaller and more precise than the popular Cas9 protein. Its more of a scalpel than a sledgehammer, he says. In a diagnostic test, the Cas protein is programmed to find a specific target. Once it finds this target, it breaks apart a reporter molecule, which can then change the color of the solution, indicating a positive or negative test result. Cas14 is particularly useful in diagnostics, Martin says, because of its size and its ability to quickly generate a signal once it finds DNA evidence of disease.
The technology has big implications for diagnostics, Martin says. One of Mammoths current partnerships is with UCSF researcher Charles Chiu, who also sits on Mammoths scientific advisory board, to create a rapid diagnostic test for the new coronavirus that has sickened more than 6,100 people globally and killed 132.
Right now, suspected coronavirus samples are shipped to the Centers for Disease Control and Prevention, where it can take six or more hours for the test to complete. The new test will work by taking a sample from a nasal swab, putting it into a tube with the Crispr-Cas system and other chemicals, and then dipping in a color-changing strip of paper to determine whether the test result is positive or negative. The whole thing should take from one to two hours, Chiu says, and be done in a doctors clinic or an emergency room. His lab was already working on a similar diagnostic test for Lyme disease, and it was able to adapt the test quickly to the new coronavirus. Chiu says the test could be ready in a matter of weeks; the only thing holding it back is a lack of human samples with which to test the diagnostic accuracy. Chiu credits Mammoths platform for helping them create a better, faster test. There are very few if any technologies that you could use that would have the same speed, turnaround and accuracy, he says.
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Mammoth Biosciences Raises $45 Million For Crispr DiagnosticsAnd Its Tech Is Already Being Used Against Coronavirus - Forbes
CRISPR Technology Market analysis by growth, segmentation, performance, competitive strategies and forecast to 2027 – WhaTech Technology and Markets…
CRISPR Technology Market to 2027 - Global Analysis and Forecasts By Product and Services (Enzymes, Kits, Services and Others), Application (Genetic Engineering, Cell Line Engineering and Others) End User (Biotechnology & Pharmaceutical Companies, Contract Research Organizations (CROs), and Academic & Government Research Institutes); and Geography
CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) technology is a simple but powerful tool for genome editing. This tool enables life science researchers to easily edit DNA sequences and modify gene function.
It has many potential applications include correcting genetic defects, treating and preventing the spread of diseases and improving crops. By delivering the CRISPR enzyme Cas9 nuclease coupled with synthetic guide RNA (gRNA) into a cell, the cell's genome can be cut at a desired location, that allows existing genes to be removed or add new ones.
Increasing usage of CRISPR systems in microbiology, growing government and private investments on research and development of genome editing, rising prevalence of genetic disorders, and increases application of CRISPR/Cas9 technology to improve crop production drives the global CRISPR technology market. However, ethical issues associated with CRISPR and lack of skilled personnel restrain the global CRISPR technology market over the forecast period.
Download Samle PDF Of Report@www.theinsightpartners.com/sample/tech-10186
Some of the key players operating in the CRISPR Technology market include :-
Thermo Fisher Scientific Inc., Merck KGaA, Horizon Discovery Group plc, Cellecta, Inc, GeneCopoeia, Inc., New England Biolabs, OriGene Technologies, Inc., GenScript, Integrated DNA Technologies, Inc. and Agilent Technologies, Inc.
The report also includes the profiles of key CRISPR Technology companies along with their SWOT analysis and market strategies.
In addition, the report focuses on leading industry players with information such as company profiles, components and services offered, financial information of last 3 years, key development in past five years.
The "Global CRISPR Technology Market Analysis to 2027" is a specialized and in-depth study of the medical device industry with a focus on the global market trend. The report aims to provide an overview of global market with detailed market segmentation by product and services, application, end user and geography.
The global CRISPR Technology market is expected to witness high growth during the forecast period. The report provides key statistics on the market status of the leading CRISPR Technology market players and offers key trends and opportunities in the market.
The global CRISPR technology market is segmented on the basis of product and services, application, end user. Based product and services, the market is segmented as, enzymes, kits, services and others.
The CRISPR technology market is categorized based on application into, genetic engineering, cell line engineering and others. Based on end user, the CRISPR Technology market is classified into biotechnology & pharmaceutical companies, contract research organizations (CROS), and academic & government research institutes.
The report provides a detailed overview of the industry including both qualitative and quantitative information. It provides overview and forecast of the global CRISPR Technology market based product and services, application, end user.
It also provides market size and forecast till 2027 for overall market with respect to five major regions, namely; North America, Europe, Asia-Pacific (APAC), Middle East and Africa (MEA) and South & Central America. The CRISPR Technology Market by each region is later sub-segmented by respective countries and segments.
The report covers analysis and forecast of 13 countries globally along with current trend and opportunities prevailing in the region.
North America held over major share in the CRISPR Technology market in 2017 owing to significant research carried out in order to develop novel therapeutics for disease targeting and high adoption of genome editing technique for germline modifications. North America is expected to collectively contribute towards the growth of CRISPR Technology market owing to the presence of major market players and also the development of technologically advanced products of CRISPR technology is expected to influence the CRISPR technology market growth.
The Asia-Pacific region is expected to exhibit highest CAGR during the forecast period due to many applications in developing economies of the region for animal disease and human disease treatment. Also, the rapid economic growth in this region coupled with diversified population and large patient pool, drives CRISPR Technology market in this region.
The report analyzes factors affecting CRISPR Technology market from both demand and supply side and further evaluates market dynamics effecting the market during the forecast period i.e., drivers, restraints, opportunities, and future trend. The report also provides exhaustive PEST analysis for all five regions namely; North America, Europe, APAC, MEA and South & Central America after evaluating political, economic, social and technological factors effecting the CRISPR Technology market in these regions.
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On The Frontlines Of Autism Research: North Carolina Professors Study Early Detection, Treatment – Here And Now
Researchers at the University of North Carolina at Chapel Hill say they can detect autism spectrum disorder before it manifests in some young children, and theyre even developing treatments for some of the conditions that go hand-in-hand with autism.
Professors Mark Zylka and Joe Piven work among more than two dozen scientists at UNC focused on autism spectrum disorder, and the National Institutes of Health have given the two more than $15 million combined in the last year alone.
Autism is a developmental disorder that affects communication and behavior, with symptoms that can include repetitive behavior and difficulty interacting with other people.
Piven has been able to detect autism in children as young as six months. And Zylka has focused on treating a syndrome closely linked to autism with gene editing, research he says could open the door to a much broader slate of treatments.
There's been a real revolution in the past 10 years in terms of our understanding of the genetic basis for autism, says Zylka, who studies cell biology and physiology. This revolution has really been sparked by the rapidly reducing cost of sequencing human genomes.
Just this month, the largest genetic study of autism to date found more than 100 genes linked to the disorder. Those kinds of breakthroughs are stepping stones toward a better understanding of autism spectrum disorder, the researchers say.
In addition to genetics, environmental factors including maternal health and prenatal exposure to air pollution play a role in the development of autism. The disorder is diagnosed in one in every 59 children in the United States, according to the Centers for Disease Control and Prevention. Two decades ago, the rate of diagnosis was just 1 in 150 children.
Piven, who studies psychiatry and pediatrics, says its hard to tell why the prevalence is increasing, though better recognition and widening criteria likely play a large role. But the increased prevalence has also generated enthusiasm for public and private funding, he says.
Earlier Is Better
With that funding including a $9.5 million grant from the NIH last year Piven and his team have been looking at the brains of kids six to 12 months old.
He uses MRI brain-imaging to predict whether a child will develop autism, well before children turn two or three and start showing symptoms. The kids hes studying have older siblings diagnosed with autism, so they run a much higher risk than the average child of developing it themselves about a 20% likelihood.
But babies who later develop autism spectrum disorder don't look like they have autism in the first year of life, he says. That's really quite amazing. And that window gives us an opportunity to think about early detection.
While the infants dont exhibit symptoms of autism, their brains look different from children who dont develop the disorder, Piven says, including differences in surface anatomy, surface area, convolutions on the surface.
Those variations have allowed him to correctly identify eight out of 10 kids who would go on to develop autism in previous studies. He says that predictive tool could allow researchers to develop early interventions.
Earlier is better, he says. As a rule of thumb in medicine, we treat things before they happen. ... We are interested in high blood pressure because it leads to stroke, so we treat high blood pressure. And that's a well-worn path.
The interventions themselves are still an open question, he says, because researchers haven't been able to identify these children in infancy before.
Turning Genes On And Off
One potential treatment, though, is gene editing.
Thats where Zylka comes in. His research, also funded by the NIH, involves mice instead of children for now. Hes trying to treat Angelman syndrome, a rare neurodevelopmental disorder often placed on the autism spectrum.
These are children that are largely non-verbal, Zylka says. They have motor problems. Its severely disabling.
People with Angelman syndrome have a mutation in the maternal UBE3A gene. Normally, any given gene passed down from one parent doesnt have to function perfectly because theres a backup the other parents gene.
But the paternal UBE3A gene is largely inactive, or silenced. Thats fine for most of us, but it means theres no backup for a child born with a missing or defective maternal gene.
The paternal gene is functional, but turned off, Zylka says. Using these new genome editing technologies like CRISPR-Cas, we're going in and trying to turn on that dad's copy of the gene.
CRISPR has gained international acclaim for its promise in treating disease, but it has also generated controversy. A Chinese researcher who said he had illegally created the world's first gene-edited babies was sentenced to prison last year.
But Zylka says his team has figured out ways to harness the tools power to treat Angelman syndrome without creating mutations that could be passed onto future generations.
So far, hes been able to treat symptoms in mice, though not eradicate them and he says the treatment has to be administered early in life to work properly. Eventually, he hopes to use CRISPR-Cas9 to edit the UBE3A genes of prenatal infants or newborns.
Zylka says hes not finding a cure per se an idea that many people with autism and advocates oppose but trying to treat potentially devastating symptoms: Angelman syndrome can cause epilepsy and severe speech impairment.
If you have a baby and at birth, they have some problem that surgery can correct, people are not going to neglect the surgery to fix the baby, he says. With genetics, we can actually pick these mutations up early, so ... gene editing approaches could be used to treat early.
He says this work could open the door to treating autism more broadly.
CRISPR-Cas technology can be used to turn genes off or turn genes on, Zylka says. Since many cases of autism are due to loss of one copy, you still have a second copy that is functional. And so you could use an editing approach to turn on the functional copy to a higher level.
Both Piven and Zylka say public funding from the NIH is a mainstay for their work. And theyre optimistic about the future of autism research, especially as more comes to light about the causes of autism syndrome disorder and the variations within that diagnosis.
While we call this autism ... these really aren't [all] the same condition, says Piven. We just have these crude behavioral criteria. So I think we will pick away at the whole and start being very successful with some that have these more simple mechanisms. And others that are more complicated, we'll have to tackle in other ways.
Zylka says subtyping disorders might even let researchers come up with personalized treatment one day. But, he adds, were not there yet.
Find more information about recruitment for this study here.
Francesca Parisproduced and edited this interview for broadcast withKathleen McKenna. Paris also adapted it for the web.
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On The Frontlines Of Autism Research: North Carolina Professors Study Early Detection, Treatment - Here And Now
DNA sleuths read the coronavirus genome, tracing its origins – STAT
As infectious disease specialists and epidemiologists race to contain the outbreak of the novel coronavirus centered on Wuhan, China, theyre getting backup thats been possible only since the explosion in genetic technologies: a deep-dive into the genome of the virus known as 2019-nCoV.
Analyses of the viral genome are already providing clues to the origins of the outbreak and even possible ways to treat the infection, a need that is becoming more urgent by the day: Early on Saturday in China, health officials reported 15 new fatalities in a single day, bringing the death toll to 41. There are now nearly 1,100 confirmed cases there.
Reading the genome (which is made of RNA, not DNA) also allows researchers to monitor how 2019-nCoV is changing and provides a roadmap for developing a diagnostic test and a vaccine.
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The genetics can tell us the true timing of the first cases and whether they occurred earlier than officials realized, said molecular biologist Kristian Andersen of Scripps Research, an expert on viral genomes. It can also tell us how the outbreak started from a single event of a virus jumping from an infected animal to a person or from a lot of animals being infected. And the genetics can tell us whats sustaining the outbreak new introductions from animals or human-to-human transmission.
Scientists in China sequenced the viruss genome and made it available on Jan. 10, just a month after the Dec. 8 report of the first case of pneumonia from an unknown virus in Wuhan. In contrast, after the SARS outbreak began in late 2002, it took scientists much longer to sequence that coronavirus. It peaked in February 2003 and the complete genome of 29,727 nucleotides wasnt sequenced until that April.
Since the sequencing of the first 2019-nCoV sample, from an early patient, scientists have completed nearly two dozen more, said Andrew Rambaut of the University of Edinburgh, an expert on viral evolution. That pace is unprecedented and completely unbelievable, said Andersen, who worked on sequencing the Ebola genome during the 2014 outbreak. Its just insane.
The genome of the Wuhan virus is 29,903 bases long, one of many clues that have led scientists to believe it is very similar to SARS.
By comparing the two dozen genomes, scientists can address the when did this start question. The 24 available samples, including from Thailand and Shenzhen as well as Wuhan, show very limited genetic variation, Rambaut concluded on an online discussion forum where virologists have been sharing data and analyses. This is indicative of a relatively recent common ancestor for all these viruses.
Given whats known about the pace at which viral genomes mutate, if nCoV had been circulating in humans since significantly before the first case was reported on Dec. 8, the 24 genomes would differ more. Applying ballpark rates of viral evolution, Rambaut estimates that the Adam (or Eve) virus from which all others are descended first appeared no earlier than Oct. 30, 2019, and no later than Nov. 29.
The progenitor virus itself was almost certainly one that circulates harmlessly in bats (as SARS does) but has an intermediate reservoir in one or more animals that come into contact with people, Andersen said. Presumably, that reservoir is one of the species of animals at the Wuhan market thought to be ground zero for the outbreak. The ancestor of 2019-nCoV existed in that species for some unknown time, never infecting people, until by chance a single virus acquired a mutation that made it capable of jumping into and infecting humans.
The genome sequences suggest that was a one-time-only jump. The genomes [from the 24 samples] are very uniform, Andersen said. If there had been multiple introductions, including from many different animals, there would be more genomic diversity. This was a single introduction.
That means that whats sustaining the spread is human-to-human transmission (suggesting that closing Wuhans animal market is very much an after-the-horse-has-fled-the-barn reaction).
Unfortunately, genetic analysis cant identify what animal species the coronavirus jumped from into humans. But an analysis by a team from the Wuhan Institute of Virology, posted to the preprint server bioRxiv, determined that the genome of this coronavirus (the seventh known to infect humans) is 96% identical to that of a bat coronavirus, suggesting that species is the original source. (Writing in the New England Journal of Medicine on Friday, another team of scientists in China reported that the new coronavirus is 86.9% identical to the bat SARS-like coronavirus.)
Virologists differ on whether its possible to read out viral properties from just the genome sequence, such as whether the microbe is spread by coughing, sneezing, touching,or merely breathing. But the analysis by the Wuhan Institute team found that it enters human cells using the same doorway that SARS did. Called angiotensin converting enzyme 2 (ACE2), the door is a receptor to which a spike protein on the viruss surface first attaches and then enables the virus to fuse with the host cell.
If ACE2 is druggable, blocking it could conceivably treat 2019-nCoV. It should be expected and worth to test if ACE2 targeting drugs can be used for nCoV-2019 patients, the scientists wrote.
The genome sequences have more to give. They will be crucially important for development of diagnostics [and] vaccines, said biologist Richard Ebright of Rutgers University.
For instance, the genome-editing technology CRISPR is the basis for Cambridge, Mass.-based startup Sherlock Biosciences diagnostics, which promise to slash how long it takes to make a definitive identification. In the U.S, thats now done only by sending samples to the Centers for Disease Control and Prevention, which uses a technology invented in the 1980s, polymerase chain reaction or PCR, to identify the presence of coronavirus.
Our vision is that our [CRISPR-based] SHERLOCK and INSPECTR platforms are tailor-made for outbreaks like coronavirus, said Sherlock CEO Rahul Dhanda, who declined to discuss specific plans related to coronavirus.
And as scientists keep adding 2019-nCoV genome sequences to their collection, they could get an early glimpse of whether the virus is mutating in a way that could make it more dangerous or more transmissible. You need continuous sequencing, Andersen said.
Correction: This story has been corrected to make clear that the coronavirus genome is made of RNA, not DNA.
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DNA sleuths read the coronavirus genome, tracing its origins - STAT
Genetic Literacy Project Releases Global Gene Editing Regulation Tracker and Index – Science 2.0
The non-profit science NGO Genetic Literacy Project has released its latest educational initiative, the Global Gene Editing Regulation Tracker and Index.
With the worldwide war on GMOs essentially lost by environmental lawyers, they still continue to hold back Europe but developing nations have seen through the false promises of western activists who have no solutions to poverty and food insecurity, only fear of the future. They are becoming hopeful about the future.
Thanks to CRISPR-Cas9 gene editing, non-chemical solutions to life-impacting developing nation problems such as malaria (dengue, yellow fever) mosquitoes can be developed, and governments will be scrambling to adapt a regulatory structure that meets the 21st century.
In the past, anti-science NGOs were able to successfully frame GMOs as too modern and terrifying. They had to ignore the existence of Mutagenesis, chemical and radiation baths used to create new strains of food and plant products in the lab, because those biotechnology results are considered part of an organic scheme. GMOs were different, they insisted.
So now they have to scramble to claim GMOs are different from mutagenesis and yet the same as CRISPR, even though they all share little in common beyond being ways to improve on nature.
So much information and disinformation can be confusing for the public. The new Genetic Literacy Project program summarizes gene editing regulations in each country's agriculture, medicine and gene efforts, along with what products and therapies are in development.
Most importantly for real progress, it also details the efforts by anti-science NGOs to block progress.
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Genetic Literacy Project Releases Global Gene Editing Regulation Tracker and Index - Science 2.0
Why Investors Rotating Towards Aphria (APHA), CRISPR Therapeutics AG (CRSP) – US Post News
The recent performance of Aphria (NYSE:APHA) stock in the market spoke loud and clear to investors as APHA saw more than 5.58M shares in trading volumes in the last trading session, way higher than the average trading volume of 5.58M shares by far recorded in the movement of Aphria (APHA). At the time the stock opened at the value of $5.17, making it a high for the given period, the value of the stock dropped by -7.97%. After the decrease, APHA touched a low price of $4.85, calling it a day with a closing price of $5.27, which means that the price of APHA went 4.85 below the opening price on the mentioned day.
Other indicators are hinting that the stock could reach an outstanding figure in the market share, which is currently set at 252.56M in the public float and 1.23B US dollars in market capitalization.
When it comes to the technical analysis of APHA stock, there are more than several important indicators on the companys success in the market, one of those being the Relative Strength Indicator (RSI), which can show, just as Stochastic measures, what is going on with the value of the stock beneath the data. This value may also indicate that the stock will go sideways rather than up or down, also indicating that the price could stay where it is for quite some time. When it comes to Stochastic reading, APHA stock are showing 52.63% in results, indicating that the stock is neither overbought or oversold at the moment, providing it with a neutral within Stochastic reading as well. Additionally, APHA with the present state of 200 MA appear to be indicating bearish trends within the movement of the stock in the market. While other metrics within the technical analysis are due to provide an outline into the value of APHA, the general sentiment in the market is inclined toward negative trends.
With the previous 100-day trading volume average of 931609 shares, CRISPR Therapeutics AG (CRSP) recorded a trading volume of 996420 shares, as the stock started the trading session at the value of $54.75, in the end touching the price of $53.59 after dropping by -2.12%.
CRSP stock seem to be going ahead the lowest price in the last 52 weeks with the latest change of 82.65%.Then price of CRSP also went backward in oppose to its average movements recorded in the previous 20 days. The price volatility of CRSP stock during the period of the last months recorded 4.56%, whilst it changed for the week, now showing 4.30% of volatility in the last seven days. The trading distance for this period is set at -10.90% and is presently away from its moving average by -15.44% in the last 50 days. During the period of the last 5 days, CRSP stock lost around -8.13% of its value, now recording a sink by 9.89% reaching an average $48.84 in the period of the last 200 days.During the period of the last 12 months, CRISPR Therapeutics AG (CRSP) dropped by -12.01%.
According to the Barcharts scale, the companys consensus rating fall to 4.27 from 4.60, showing an overall improvement during the course of a single month. Based on the latest results, analysts are suggesting that the target price for CRSP stock should be $53.59 per share in the course of the next 12 months. To achieve the target price as suggested by analysts, CRSP should have a spike by 0% in oppose to its present value in the market. Additionally, the current price showcases a discount of 48.47% when compared to the high consensus price target predicted by analysts.
CRSP shares recorded a trading volume of 834200 shares, compared to the volume of 1.22M shares before the last close, presented as its trading average. With the approaching 4.30% during the last seven days, the volatility of CRSP stock remained at 4.56%. During the last trading session, the lost value that CRSP stock recorded was set at the price of $53.59, while the lowest value in the last 52 weeks was set at $29.34. The recovery of the stock in the market has notably added 82.65% of gains since its low value, also recording -20.29% in the period of the last 1 month.
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Why Investors Rotating Towards Aphria (APHA), CRISPR Therapeutics AG (CRSP) - US Post News
After a bone injury, shape-shifting cells rush to the rescue – University of Michigan News
ANN ARBORConventional thinking is that bone regeneration is left to a small number of mighty cells called skeletal stem cells, which reside within larger groups of bone marrow stromal cells.
But new findings from the University of Michigan recasts that thinking.
In a recent study, Noriaki Ono, assistant professor at the U-M School of Dentistry, and colleagues report that mature bone marrow stromal cells metamorphosed to perform in ways similar to their bone-healing stem cell cousinsbut only after an injury.
Bone fracture is an emergency for humans and all vertebrates, so the sooner cells start the business of healing damaged boneand the more cells there are to do itthe better.
Our study shows that other cells besides skeletal stem cells can do this job as well, Ono said.
In the mouse study, inert Cxcl12 cells in bone marrow responded to post-injury cellular cues by converting into regenerative cells, much like skeletal stem cells. Normally, the main job of these Cxcl12-expressing cells, widely known as CAR cells, is to secrete cytokines, which help regulate neighboring blood cells. They were recruited for healing only after an injury.
The surprise in our study is that these cells essentially did nothing in terms of making bones, when bones grow longer, Ono said. Its only when bones are injured that these cells start rushing to repair the defect.
This is important because the remarkable regenerative potential of bones is generally attributed to rare skeletal stem cells, Ono says. These new findings raise the possibility that these mighty skeletal stem cells could be generated through the transformation of the more available mature stromal cells.
These mature stromal cells are malleable and readily available throughout life, and could potentially provide an excellent cellular source for bone and tissue regeneration, Ono says.
The study appears in the journal Nature Communications.
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After a bone injury, shape-shifting cells rush to the rescue - University of Michigan News
What are Progenitor Cells? Exploring Neural, Myeloid and Hematopoietic Progenitor Cells – Technology Networks
What are progenitor cells?
Every cell in the human body, and that of other mammals, originates from stem cell precursors. Progenitor cells are descendants of stem cells that then further differentiate to create specialized cell types.There are many types of progenitor cells throughout the human body. Each progenitor cell is only capable of differentiating into cells that belong to the same tissue or organ. Some progenitor cells have one final target cell that they differentiate to, while others have the potential to terminate in more than one cell type.
Stem cells share two qualifying characteristics. Firstly, all stem cells have the potential to differentiate into multiple types of cells. Secondly, stem cells are capable of unlimited self-replication via asymmetric cell division, a process known as self-renewal.There are two broad categories of stem cells found in all mammals. The first are embryonic stem cells. These cells arise from the inner cell mass of the blastocyst in an early-stage embryo. Embryonic stem cells are the blueprint used to create every cell in the body. Because they can be used to create any type of cell, they are known as pluripotent.
The second type of stem cells found in mammals are adult stem cells (or somatic stem cells). Unlike pluripotent embryonic stem cells, adult stem cells are more limited in relation to the type of cells that they become. Unlike embryonic stem cells that could be used to create any cell, adult stem cells are limited to generating cell types within a specific lineage, such as blood cells or cells of the central nervous system. This level of differentiation potential is termed multipotent.
Stem cells create two types of progeny: more stem cells or progenitor cells. All progenitor cells are descendants of stem cells. When it comes to cell differentiation, they fall on the spectrum between stem cells and fully differentiated (mature) cells.
Whilst stem cells have indefinite replication (left) progenitor cells can at most differentiate into multiple types of specialized cell (right).
Function:
Cellular repair or maintenance
Cell Potency:
Multipotent, oligopotent, or unipotent
Self-renewal:
Limited
Origin:
Stem cells
Creates:
Further differentiated cells (either progenitor cells of mature/fully differentiated cells)
Progenitor cells are an intermediary step involved in the creation of mature cells in human tissues and organs, the blood, and the central nervous system.
The human central nervous system (CNS) contains three types of fully differentiated cells: neurons, astrocytes and oligodendrocytes. The latter two are collectively known as glial cells.Every neuron, oligodendrocyte and astrocyte in the CNS evolves from the differentiation of neural progenitor cells (NPCs). NPCs themselves are produced by multipotent neural stem cells (NSCs). Both NPCs and NSCs are termed neural precursor cells.Before the 1990s, it was believed that neurogenesis terminated early in life. More recent studies demonstrate that the brain contains stem cells that are capable of regenerating neurons and glial cells throughout the human lifecycle. These stem cells have only been found in certain brain regions, including the striatum and lateral ventricle.
Hematopoietic progenitor cells (HPCs) are an intermediate cell type in blood cell development. HPCs are immature cells that develop from hematopoietic stem cells, cells that can both self-renew and differentiate into hematopoietic progenitor cells. HPCs eventually differentiate into one of more than ten different types of mature blood cells.Hematopoietic progenitor cells are categorized based upon their cell potency, or their differentiation potential. As blood cells develop, their potency decreases.
First, hematopoietic stem cells differentiate into multipotent progenitor cells. Multipotent progenitor cells are those with the potential to differentiate into a subset of cell types. These cells then differentiate into either the common myeloid progenitor (CMP) or common lymphoid progenitor (CLP). Both CMPs and CLPs are types of oligopotent progenitor cells (progenitor cells that differentiate into only a few cell types).
CMPs and CLPs continue to differentiate along cell lines into lineage-restricted progenitor cells that become final, mature blood cells.Myeloid progenitor cells are precursors to the following types of blood cells:
Lymphoid progenitor cells (also known as lymphoblasts) are precursors to other mature blood cell types, including:
The primary role of progenitor cells is to replace dead or damaged cells. In this way, progenitor cells are necessary for repair after injury and as part of ongoing tissue maintenance. Progenitor cells also replenish blood cells and play a role in embryonic development.
Neural progenitor cells (NPCs) are being explored alongside neural stem cells for their potential to treat diseases of or injury to the central nervous system. A deeper understanding of how these cells function on a cellular and molecular basis is needed to progress from early experimental research to therapeutic use.NPCs are currently utilized in research conducted on CNS disorders, development, cell regeneration and degeneration, neuronal excitability, and therapy screening. When compared to induced pluripotent stem cells, which are cells reprogrammed into a pluripotent state, NPCs can cut down on time in some experiments.Hematopoietic progenitor cells and stem cells are being researched for their capacity to treat blood cell disorders. They are also currently used to help treat patients with a variety of malignant and non-malignant diseases via bone marrow transplants that deliver bone marrow and peripheral blood progenitor cells to patients. These procedures can assist patients in recovering from the damage caused by chemotherapy.Additionally, researchers are examining the potential of using progenitor cells to create a variety of tissues, such as blood vessels, heart valves, and electrically conductive tissue for the cardiovascular system.
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What are Progenitor Cells? Exploring Neural, Myeloid and Hematopoietic Progenitor Cells - Technology Networks
Pain treatment using human stem cells a success – News – The University of Sydney
Chronic pain cost an estimated $139.3 billion in 2018. Image: iStock, Top image: Pexels
Researchers at the University of Sydney have used human stem cells to make pain-killing neurons that provide lasting relief in mice, without side effects, in a single treatment. The next step is to perform extensive safety tests in rodents and pigs, and then move to human patients suffering chronic pain within the next five years.
If the tests are successful in humans, it could be a major breakthrough in the development of new non-opioid, non-addictive pain management strategies for patients, the researchers said.
Thanks to funding from the NSW Ministry of Health, we are already moving towards testing in humans, said Professor Greg Neely, a leader in pain research at the Charles Perkins Centre and the School of Life and Environmental Sciences.
Nerve injury can lead to devastating neuropathic pain and for the majority of patients there are no effective therapies. This breakthrough means for some of these patients, we could make pain-killing transplants from their own cells, and the cells can then reverse the underlying cause of pain.
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Pain treatment using human stem cells a success - News - The University of Sydney
Watch | Stem cell banking and its benefits – The Hindu
The blood collected from the umbilical cord of the newborn is a rich source of stem cells. This blood is collected and sent to a cord blood bank, where the stem cells are separated, tested, processed, and preserved in liquid nitrogen. Technically, there is no expiry date and these stem cells can be preserved for a lifetime. Scientifically, evidence exists that they can be stored for about 20 years. The stem cells can treat around 70 blood related disorders and genetic disorders including thalassemia, sickle cell anaemia, leukaemia, and immune related disorders.
Stem cells taken from umbilical cord blood are like those taken from bone marrow, capable of producing all blood cells: red cells, platelets and immune system cells. When used, stem cells are first concentrated, then injected into the patient. Once transfused, they produce new cells of every kind.
They're capable of producing all types of blood cells: red cells, platelets and immune system cells. The stem cells can treat around 80 blood related illnesses like leukaemia, lymphomas, several genetic conditions and immune related disorders. But given the present state of medicine, they are effective only for around a dozen of them
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Watch | Stem cell banking and its benefits - The Hindu
Cancer patient flies dad who saved his life 6600 miles away around the world – Birmingham Live
A dad who saved the life of a cancer patient 6,600 miles away was flown around the world on a trip of a lifetime by his grateful recipient - who tracked him down.
Milton Becker, 69, was close to death and in desperate need of a bone marrow donor when a two-and-a-half year global search linked him with an anonymous Welsh man.
Emyr Williams, 54, was a near-perfect match, and his bone marrow was flown to Canada and given to Milton, who was declared cancer free.
The pair were linked up by the donation database and grew close via phone calls and Facebook messages.
And last year he invited retired carpenter Emyr to Canada for a two week body">
A dad who saved the life of a cancer patient 6,600 miles away was flown around the world on a trip of a lifetime by his grateful recipient - who tracked him down.
Milton Becker, 69, was close to death and in desperate need of a bone marrow donor when a two-and-a-half year global search linked him with an anonymous Welsh man.
Emyr Williams, 54, was a near-perfect match, and his bone marrow was flown to Canada and given to Milton, who was declared cancer free.
The pair were linked up by the donation database and grew close via phone calls and Facebook messages.
And last year he invited retired carpenter Emyr to Canada for a two week $15,000 (8,835) trip around Alberta and the Rocky Mountains.
Meeting him for the first time at the airport, wearing a Welsh dragon T-shirt and a Wales flag, they formed an instant bond.
Milton said he's "indebted" to his hero - and is planning a UK trip.
Dad-of-three Emyr, from Lampeter, Wales, said: "It was surreal to be out there.
"There's this bond between us like no other.
"It was only when we went out there that we really understood how close to death Milton was.
"One of his friends said he had been finalising plans to be at his funeral.
"He was literally on death's door.
"For something that required no real effort at all saved that great man's life.
"And to have the pleasure of meeting him in the flesh and to be introduced to his family was just an honour."
Granddad-of-two Milton, from Alberta, Canada, added: "We got on so well and I just thought I've got to thank this guy.
"I didn't want him to spend a penny. It was my treat.
"It's not about the money. What he did was priceless.
"I'm forever indebted to the guy."
Milton was diagnosed with stage 4 leukaemia in 2010 but after unsuccessful chemotherapy he was told a bone marrow transplant was the only means of survival.
Doctors searched across Canada but were unsuccessful and begun their two-and-a-half year worldwide search for a donor.
In early 2013, Emyr - who had been registered on the blood transfusion register for several years - was found to be a near-perfect match.
Emyr said: "A lady called me one day to say 'would you be interested in donating your stem cells?
"She went on to say there was a guy in Canada with leukaemia and that I was a 99.9999 per cent match with him.
"I just thought why not.
"It doesn't cost me anything and it can really change somebody's life."
The bone marrow was flown from Wales - with Milton receiving his long-awaited transfusion on his 63rd birthday, on 1st February 2013.
Former oil company lorry driver Milton said: "What he did was completely priceless.
"There's no better gift than the gift of life.
"And to get that on my birthday, well, it was a great feeling!"
A year after the transfusion Milton was told he was on the road to recovery but was kept in remission and monitored by doctors for the next two years.
In 2016, three years after the blood transfusion, Milton was deemed cancer-free.
It led nurses to ask Milton if he would like to know who his donor was - which he accepted straight away.
They got in touch with Emyr - who'd been given bi-annual anonymous updates - who agreed his details could be passed on.
Emyr said: "A few days later I had this call from an international number.
"I remember it as clear as day.
"He phoned me up and said; 'Emyr, my name is Milton and I just want to say how thankful I am'.
"From then on we just hit it off.
"What makes me laugh is he always forgets his Facebook password so he's a complete technophobe.
"We speak through his children on Facebook.
"We mostly speak about our family."
Milton said: "I couldn't turn up the chance to thank the guy who gave me life!
"I started off by thanking him and we had a great chat.
"I told him I would be forever grateful and wanted to keep in touch."
The two then added each other on Facebook and soon became good friends with weekly messages and monthly phone calls.
Then two years later Milton phoned Emyr to ask if he and his family would be interested in flying out to Canada for a two-week holiday.
Emyr said: "He asked me during one of our phone calls.
"I had never been to Canada and thought it would just be great to meet each other face-to-face."
Emyr flew out with his wife and teenage daughter last September 2019 to start the two-week itinerary around Alberta and the Rocky Mountains.
Emyr said: "He was there at the airport with a Welsh dragon on his T-shirt and a Welsh flag.
"You couldn't miss them.
"We have beautiful mountains here in Wales but Canada was just something else.
"It was an absolutely incredible trip.
"He paid for it all.
"We stayed in cabins, had a party with his extended family, we drank, sat by the open fire, and toasted marshmallows."
Milton added: "One Sunday I took him to my church.
"People knew he was coming and the service and to my surprise Emyr got up and told the church about the successful operation.
"There were tears but it was just beautiful."
Now seven years on from the transfusion, the pair say they are thankful to have one another in each other's lives.
The pair still keep regular contact with one another, with Facebook messages, fortnightly phone calls and even FaceTimed each other on Christmas Day.
Emyr said: "They're planning on coming to Wales next year in June or July.
"We'll definitely go back out there again in a few years.
"Even though we're thousands of miles away, we're such great friends."
Milton said: "We still have our chit-chats and I'd love to go over to the UK.
5,000 (8,835) trip around Alberta and the Rocky Mountains.
Meeting him for the first time at the airport, wearing a Welsh dragon T-shirt and a Wales flag, they formed an instant bond.
Milton said he's "indebted" to his hero - and is planning a UK trip.
Dad-of-three Emyr, from Lampeter, Wales, said: "It was surreal to be out there.
"There's this bond between us like no other.
"It was only when we went out there that we really understood how close to death Milton was.
"One of his friends said he had been finalising plans to be at his funeral.
"He was literally on death's door.
"For something that required no real effort at all saved that great man's life.
"And to have the pleasure of meeting him in the flesh and to be introduced to his family was just an honour."
Granddad-of-two Milton, from Alberta, Canada, added: "We got on so well and I just thought I've got to thank this guy.
"I didn't want him to spend a penny. It was my treat.
"It's not about the money. What he did was priceless.
"I'm forever indebted to the guy."
Milton was diagnosed with stage 4 leukaemia in 2010 but after unsuccessful chemotherapy he was told a bone marrow transplant was the only means of survival.
Doctors searched across Canada but were unsuccessful and begun their two-and-a-half year worldwide search for a donor.
In early 2013, Emyr - who had been registered on the blood transfusion register for several years - was found to be a near-perfect match.
Emyr said: "A lady called me one day to say 'would you be interested in donating your stem cells?
"She went on to say there was a guy in Canada with leukaemia and that I was a 99.9999 per cent match with him.
"I just thought why not.
"It doesn't cost me anything and it can really change somebody's life."
The bone marrow was flown from Wales - with Milton receiving his long-awaited transfusion on his 63rd birthday, on 1st February 2013.
Former oil company lorry driver Milton said: "What he did was completely priceless.
"There's no better gift than the gift of life.
"And to get that on my birthday, well, it was a great feeling!"
A year after the transfusion Milton was told he was on the road to recovery but was kept in remission and monitored by doctors for the next two years.
In 2016, three years after the blood transfusion, Milton was deemed cancer-free.
It led nurses to ask Milton if he would like to know who his donor was - which he accepted straight away.
They got in touch with Emyr - who'd been given bi-annual anonymous updates - who agreed his details could be passed on.
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Cancer patient flies dad who saved his life 6600 miles away around the world - Birmingham Live
Umbilical Cord Blood Banking Market By Classifications, Applications and Market Overview 2019-2027 – NY Telecast 99
Umbilical cord blood banking or cord blood banking is the practice of preserving blood from the umbilical cord for future use. Such preserved cord blood is used in medical therapies in the similar approach as that of stem cells derived from bone marrow. Umbilical cord blood is collected from the umbilical cord of a newborn baby and also retrieved from the placenta after delivery. It is enriched with adult stem cells and these stem cells play a vital role in regulating all biological activities and in developing tissues in the human body.
The market of umbilical cord blood banking is anticipated to grow with a significant rate in the coming years, owing to factors such as, increasing prevalence of chronic diseases is the key driver of the umbilical cord blood banking market. Globally, umbilical cord blood banking market is growing rapidly due to, various government associations and initiatives are also supporting the growth of the market. Asia Pacific region are expected to offer growth opportunities for the players operating in the market owing to increasing prevalence of chronic diseases.
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The global umbilical cord blood banking market is segmented on the basis of product, application, and end users. The product segment includes, public cord blood banks, and private cord blood banks. The umbilical cord blood banking market based on the application is segmented as, cancer, blood disorders, metabolic disorders, immune disorders, osteoporosis and, others application. Based on the end users, the umbilical cord blood banking market is segmented as, hospitals, pharmaceutical research and, research institutes.
The report provides a detailed overview of the industry including both qualitative and quantitative information. It provides overview and forecast of the global umbilical cord blood banking market based on product, application, and end users. It also provides market size and forecast till 2027 for overall Umbilical cord blood banking market with respect to five major regions, namely; North America, Europe, Asia-Pacific (APAC), Middle East and Africa (MEA) and South & Central America. The market by each region is later sub-segmented by respective countries and segments. The report covers analysis and forecast of 13 counties globally along with current trend and opportunities prevailing in the region.
North America holds the largest share for umbilical cord blood banking market. This largest share of the region can be attributed to increasing prevalence of chronic diseases and rising awareness about importance of cord blood. However, Asia Pacific is the fastest growing region in the umbilical cord blood banking market over the forecast period. Although the region currently holds a nominal share in the global market, it offers enormous growth potential owing to vast improvement in health care reforms and increasing awareness of stem cell banking in selected countries of Asia Pacific, such as India, China, and Japan.
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The Benefit of Adding Daratumumab to Multiple Myeloma Drug Combinations – SurvivorNet
Daratumumab: A Promising Option
Due to promising new data from several big clinical trials, its now believed that daratumumab can benefit patients with multiple myeloma regardless ofwhether theyre eligible to receive a stem cell transplant.
Daratumumab (also known by its brand name, Darzalex), is a type of drug called a targeted monoclonal antibody. It works by binding to a specific protein called CD38, which is found on the surface of multiple myeloma cells. Once the daratumumab attaches to these proteins on the surface of the cells, the bodys immune system identifies the need to attack and kill the multiple myeloma cells.
As Dr. Nina Shah,a hematologist at the University of California San Francisco, explains, patients receiving a stem cell transplant can benefit from the addition of daratumumab to a combination of the drugs Velcade, Revlimid and dexamethasone (a combination that doctors often abbreviate as Dara VRD).
If, on the other hand, a transplant isnt the right course of treatment for you, you may still be able to benefit from daratumumab when its used in whats called the upfront or first-line treatment setting (meaning as the first part of your treatment, before you receive other drugs) when combined withRevlimid and dexamethasone (a combination that doctors often abbreviate as DRD).
If youre not interested in a transplant, or maybe thats not in the works for you at the moment, you may consider daratumumab, Revlimid and dexamethasone, or DRD,' Dr. Shah explains.
One thing thats really gotten a lot of attention is not just three drugs, but four drugs, Dr. Shah says. She explains that a recent clinical trial called GRIFFIN showed that before a stem cell transplant, treatment with daratumumab in combination with Velcade, Revlimid and dexamethasone was more beneficial than treatment with Velcade, Revlimid and dexamethasone alone.
And when patients who received this combination before their transplant, then went on to receive additional daratumumab after their transplant, the benefit was even greater.
Preliminarily, at least, the data seems to indicate that the patients who got four drugs, then went on to a transplant and then got more daratumumab actually did better than the three drugs, Dr. Shah says.
Learn more about SurvivorNet's rigorous medical review process.
Dr. Nina Shah is a hematologist who specializes in the treatment of multiple myeloma, a type of cancer affecting the blood marrow. She treats patients at the Hematology and Blood and Marrow Transplant Clinic. Read More
Daratumumab (also known by its brand name, Darzalex), is a type of drug called a targeted monoclonal antibody. It works by binding to a specific protein called CD38, which is found on the surface of multiple myeloma cells. Once the daratumumab attaches to these proteins on the surface of the cells, the bodys immune system identifies the need to attack and kill the multiple myeloma cells.
If, on the other hand, a transplant isnt the right course of treatment for you, you may still be able to benefit from daratumumab when its used in whats called the upfront or first-line treatment setting (meaning as the first part of your treatment, before you receive other drugs) when combined withRevlimid and dexamethasone (a combination that doctors often abbreviate as DRD).
If youre not interested in a transplant, or maybe thats not in the works for you at the moment, you may consider daratumumab, Revlimid and dexamethasone, or DRD,' Dr. Shah explains.
One thing thats really gotten a lot of attention is not just three drugs, but four drugs, Dr. Shah says. She explains that a recent clinical trial called GRIFFIN showed that before a stem cell transplant, treatment with daratumumab in combination with Velcade, Revlimid and dexamethasone was more beneficial than treatment with Velcade, Revlimid and dexamethasone alone.
And when patients who received this combination before their transplant, then went on to receive additional daratumumab after their transplant, the benefit was even greater.
Preliminarily, at least, the data seems to indicate that the patients who got four drugs, then went on to a transplant and then got more daratumumab actually did better than the three drugs, Dr. Shah says.
Learn more about SurvivorNet's rigorous medical review process.
Dr. Nina Shah is a hematologist who specializes in the treatment of multiple myeloma, a type of cancer affecting the blood marrow. She treats patients at the Hematology and Blood and Marrow Transplant Clinic. Read More
Excerpt from:
The Benefit of Adding Daratumumab to Multiple Myeloma Drug Combinations - SurvivorNet
Novel Drug May Reverse Breast Cancer Hormone Therapy Resistance – Technology Networks
A novel drug based on a natural compound found in broccoli, kale and other cruciferous vegetables could hold the key to reversing or even preventing resistance to breast cancer hormone therapy, new research has found.Scientists from the University of Manchester found that drug SFX-01 which has shown promise in a phase II trial (STEM) as a treatment for secondary breast cancer that is already resistant to hormone therapy could reverse or even prevent resistance to hormone therapy by blocking a key cancer signaling pathway (a chain of reactions within cancer cells) called STAT3.
Breast cancer is the UKs most common cancer, with around 55,000 women and approximately 370 men being diagnosed throughout the country each year.
Up to 80% of breast cancers are encouraged to grow by the hormone estrogen and are known as estrogen receptor (ER) positive breast cancer, which accounts for up to 44,000 cases each year in the UK.
While hormone therapy (which blocks the effect of estrogen) is very effective in reducing the risk of recurrence for most, around a third of patients with ER positive breast cancer see their disease return within 15 years, and some of these are due to the cancer developing resistance to treatment.
SFX-01 inspired by a natural plant-derived compound called sulforaphane, which was first discovered in cruciferous vegetables such as rocket, broccoli and kale has recently been shown in a clinical trial to delay the progression of incurable secondary breast cancer in women whose disease has already developed resistance to hormone therapy.
In a new study led by Dr Bruno Simes, Dr Sacha Howell and Professor Rob Clarke at the University of Manchester, researchers investigated the effect of SFX-01 alone, or in combination with tamoxifen or fulvestrant, in patient samples and in mice to understand how the drug works and how it can be best used to treat breast cancer.
They found that SFX-01 reduced the ability of specialized cells called breast cancer stem cells to form tumors in mice, with the drug also reducing the ability of breast cancer cells to form secondary tumors in the mices lungs.
The researchers then looked at the gene activity levels within the breast cancer stem cells from hormone therapy-resistant tumor samples from patients, finding that the cancer stem cells relied heavily on the STAT3 signaling pathway, which can become active in response to hormone therapy and lead to treatment resistance.
SFX-01 blocked the STAT3 signaling pathway and reversed the effects that may lead to hormone therapy resistance.
In a recent phase II trial (STEM) in patients with ER positive secondary breast cancer that had already started becoming resistant to hormone therapy, 25% of participants benefitted from the addition of SFX-01 to hormone therapybut the mechanism of why this worked was not known until now.
Further research is now focusing on understanding why certain patients tumors are sensitive to SFX-01 and whether an accompanying diagnostic test for activity of the STAT3 signaling pathway could be used to identify the patients that would benefit the most from this treatment.
It is also hoped that SFX-01 could in future be added to hormone therapies such as tamoxifen or aromatase inhibitors from the outset of treatment to increase their effectiveness in patients with primary breast cancer.
Co-author Dr Bruno Simes, Research Fellow at the University of Manchester, said:
Estrogen receptor positive breast cancer is the most common breast cancer. These cancers frequently develop resistance to hormone therapies, which is a major clinical problem that we are working to address.
We are excited by our findings that combining standard hormone therapies with SFX-01 could improve treatment of some breast cancer patients by reversing resistance driven by the STAT3 signaling pathway.
With the success of the recent clinical trial in secondary breast cancer, we hope that further studies will now help to identify which patients may benefit the most from this drug so that it could soon reach the clinic.
Dr Simon Vincent, Director of Research at Breast Cancer Now, which helped to fund the study, said:
Its really exciting that SFX-01 could in future help to improve the effectiveness of hormone therapies and prevent or treat the return of breast cancer. While hormone therapy is effective for most women, around a third still see their breast cancer return and we urgently need to find new ways to tackle and prevent drug resistance.
This important discovery reveals exactly how SFX-01 can help overcome hormone therapy resistance and we hope it could now open the door to it being used from the outset of treatment, to prevent resistance from developing in the first place.
We look forward to results of further trials to fully understand who is likely to benefit most and at what stage of treatment it should be added to hormone therapy to give patients the best chance of survival.
The study is being presented at the UK Interdisciplinary Breast Cancer Symposium, hosted by Breast Cancer Now.
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Novel Drug May Reverse Breast Cancer Hormone Therapy Resistance - Technology Networks