Archive for November, 2014

ORLANDO, Fla. -

Little Hunter Miller's motor is always running. Like most toddlers, he's sometimes one step away from trouble, but for Hunter, being rough and tumble can have serious side effects. Hunter has severe hemophilia.

Three days after he was born, a routine circumcision caused a major scare.

"You know, a baby gets up in the morning and their diapers are just full," said Hunter's grandmother, Tina Miller. "Well, his was full, but it was full of blood."

Doctors diagnosed Hunter with hemophilia A, which means his blood is missing a protein, known as clotting factor VIII. When he gets hurt, doctors need to inject the clotting factor to stop the bleeding. He's had eight emergency room visits in 19 months.

"Him falling, bumping his head too hard, little cuts. He cut the roof of his mouth with a tortilla chip and that was a hospital trip," said Heather Frederick, Hunter's mother.

Dr. Katherine Ponder studies gene therapy treatment for hemophilia and other blood disorders. Her lab treated hemophilia A in animals, but she said the therapy isn't quite ready for humans yet.

"I think that the big question is going to be the safety," said Katherine Ponder, hematologist at Washington University School of Medicine in St. Louis.

But gene therapy has proven effective for some patients with hemophilia B. Researchers at St. Jude's Children's Research Hospital and University College of London have added the missing protein -- factor IX -- to a specially-engineered virus, which travels to the patients liver and transfers the gene.

"This modifies the disease from a situation where they might bleed once a week to a situation where they hardly ever bleed," Ponder explained.

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Health Beat: Gene therapy: From bench to bedside: Hemophilia

Cambridge stem cell pioneer DefiniGEN is in China this week showcasing technology that arguably gives the UK a world lead in countering liver and pancreatic cancer.

The young company is seeking Chinese partners to broaden the reach of the technology which holds a potentially significant payback in regenerative medicine.

With US global stem cell innovator Roger Pedersen among its technology founders, DefiniGEN was founded two years ago to commercialise a stem cell production platform developed at the University of Cambridge.

The platform generates human liver and pancreatic cell types using Nobel Prize winning human Induced Pluripotent Stem Cell (iPSC) technology.

DefiniGEN is visiting Shanghai and Beijing on a trade mission organised by UKTI East of England in partnership with the China-Britain Business Council.

The company is actively looking to partner with Life Science distributors and pharmaceutical drug discovery companies in China. CEO Dr Marcus Yeo and Dr Masashi Matsunaga business development manager for Asia Pacific - are spearheading the initiative.

The visit includes a range of medically-focused ventures from one to one meetings with key players to presentations at UK consulates.

DefiniGEN cells are provided to the drug discovery sector for use in lead optimisation and toxicity programmes.

The companys OptiDIFF platform produces validated libraries of disease-modelled human liver cells for a range of diseases. The phenotype (the composite of an organisms traits) and pathology of the diseases is pre-confirmed in the cells.

The technology provides pharmaceutical companies with more predictive in vitro cell products enabling the development of safer and more effective treatments.

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Cambridge stem cell pioneer targets China partners

Chuck Bednar for redOrbit.com Your Universe Online

A new stem cell gene therapy developed by researchers at UCLA is set to begin clinical trials early next year after the technique reportedly cured 18 children who were born without working immune systems due to a condition known as ADA-deficient Severe Combined Immunodeficiency (SCID) or Bubble Baby disease.

The treatment was developed by Dr. Donald Kohn, a member of the UCLA Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research, and his colleagues, and according to the university, it is able to identify and correct faulty genes by using the DNA of the youngsters born with this life-threatening condition.

Left untreated, ADA-deficient SCID is often fatal within the first year of a childs life, reports Peter M. Bracke for UCLA. However, after more than three decades of research, Dr. Kohns team managed to develop a gene therapy that can safely restore the immune systems of children with the disease by using their own cells and with no noticeable side effects.

All of the children with SCID that I have treated in these stem cell clinical trials would have died in a year or less without this gene therapy, instead they are all thriving with fully functioning immune systems, Dr. Kohn, who is also a professor of pediatrics and of microbiology, immunology and molecular genetics, said in a recent statement.

Children born with SCID have to be isolated in a controlled environment for their own safety, because without an immune system, they are extremely vulnerable to illnesses and infections that could be deadly. While there are other treatments for ADA-deficient SCID, Dr. Kohn noted that they are not always optimal or feasible for many children. The new technique, however, provides them with a cure, and the chance to live a full healthy life.

SCID is an inherited immunodeficiency that is typically diagnosed about six months after birth, the researchers said, and children with the condition are so vulnerable to infectious diseases that even the common cold could prove fatal to them. This particular form of the condition causes cells to not create ADA, an enzyme essential for the production of the white blood cells which are a vital component of a healthy, normally-functioning immune system.

Approximately 15 percent of all SCID patients are ADA-deficient, according to the university, and these youngsters are typically treated by being injected twice per week with the required enzyme. This is a process that must continue throughout a patients entire life, and even then it doesnt always work to bring their immune systems to optimal levels. Alternately, they could undergo bone marrow transplants from matched siblings, but those matches are rare and the transplanted cells themselves are often rejected by the childs body.

Dr. Kohn and his colleagues tested two therapy regimens on 18 ADA-deficient SCID over the course of two multi-year clinical trials starting in 2009. During the trials, the blood stem cells of the patients were removed from their bone marrow and genetically modified in order to correct the defect. All 18 of the patients were cured.

The technique used a virus delivery system first developed in Dr. Kohns laboratory in the 1990s a technique which inserts the corrected gene that produces the ADA into the blood forming stem cells in the bone marrow. The genetically corrected blood-forming stem cells will then produce the T-cells required to combat infections.

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New Stem Cell Treatment Found To Cure 'Bubble Baby' Disease

1. Keratinocytes

There are two approaches to commit ES cells and adult stem cells (of non-epidermal origin) to the keratinocyte lineage in vitro. One approach would be to expose the cells to a cocktail of exogenous cytokines, growth factors, chemicals, and extracellular matrix (ECM) substrata over a prolonged duration of in vitro culture. Only a fraction of the stem cells would be expected to undergo commitment to the keratinocyte lineage, because many of these cytokines, growth factors, chemicals, and ECM substrata would exert non-specific pleitropic effects on stem cell differentiation into multiple lineages. At best, the cocktail combination of various cytokines, growth factors, chemicals, and ECM substrata can be optimized by trial and error, to maximize the proportion of stem cells committing to the keratinocyte lineage, while at the same time yielding a large number of other undesired lineages. Hence, extensive selection/purification and proliferation of the commited keratinocyte progenitors is likely to be required.

By using such an approach, Coraux et al.54 managed to achieve commitment and subsequent differentiation of murine ES cells into the keratinocyte lineage, in the presence of a cocktail combination of bone morphogenetic protein-4 (BMP-4), ascorbate, and ECM derived from human normal fibroblasts (HNFs) and murine NIH-3T3 fibroblasts. Nevertheless, it must be noted that the study of Coraux et al.54 also reported a high degree (approximately 80%) of non-specific differentiation into multiple uncharacterized lineages, and no attempt was made to purify differentiated keratinocytes or keratinocyte progenitors from the mixture of lineages derived from murine ES cells. Bagutti et al.61 reported that coculture with human dermal fibroblasts (HDFs) as well as HDF-conditioned media could induce beta integrin- deficient murine ES cells to commit and differentiate into the keratinocyte lineage. However, as with the study of Coraux et al.,54 the keratinocytes were interspersed with differentiated cells of other lineages. Recently, differentiation of human ES cells into the keratinocyte lineage was also reported by Green et al.62 However, this study was based on in vivo teratoma formation within a SCID mouse model, and to date, there are no parallel in vitro studies that have been reported.

With adult stem cells of non-epidermal origin, there are also few studies 63, 64 which have successfully achieved re-commitment and trans-differentiation to the keratinocyte lineage. Even so, these studies were based primarily on the transplantation of undifferentiated stem cells in vivo, with the observed trans-differentiation occurring sporadically and at extremely low frequencies. Moreover, the validity of the experimental data may be clouded by controversy over the artifact of stem cell fusion in vivo.65 To date, there are no parallel in vitro studies that have achieved recommitment and trans-differentiation of non-epidermal adult stem cells to the keratinocyte lineage. It can therefore be surmised that the use of exogenous cytokines, growth factors, chemicals, and ECM substrata to induce ES cell and nonepidermal adult stem cell commitment to the keratinocyte lineage is a relatively inefficient, time-consuming, and labor-intensive process that would require extensive selection and purification of the committed keratinocyte progenitors. Hence, it would be technically challenging to apply this to the clinical situation.

The other approach for inducing ES cell and non-epidermal adult stem cell commitment to the keratinocyte lineage is through genetic modulation. This may be achieved by transfecting stem cells with recombinant DNA constructs encoding for the expression of signaling proteins that promote commitment to the keratinocyte lineage. Of particular interest are the Lef-1/Tcf family of Wnt regulated transcription factors that act in concert with b-catenin,66, 67 c-myc which is a downstream target of the Wnt-signaling pathway,68, 69 and the transactivation domain containing isoform of transcription factor p63 (Tap63).70, 71 Interestingly, the transcription factor GATA-3, which is well known to be a key regulator of T-cell lineage determination, has also been shown to be essential for stem cell lineage determination in skin, where it is expressed at the onset of epidermal stratification and Inner Root Sheath (IRS) specification in follicles.72 Recombinant overexpression of p6373 and c-Myc74 has been reported to promote commitment and differentiation to the keratinocyte lineage.

The disadvantage of directing differentiation through genetic modulation is the potential risks associated with utilizing recombinant DNA technology in human clinical therapy. For example, the overexpression of any one particular protein within transfected stem cells would certainly have unpredictable physiological effects upon transplantation in vivo. This problem may be overcome by placing the recombinant expression of the particular protein under the control of switchable promoters, several of which have been developed for expression in eukaryotic systems. Such switchable promoters could be responsive to exogenous chemicals,75 heat shock,76 or even light.77 Genetically modified stem cells may also run the risk of becoming malignant within the transplanted recipient. Moreover, there are overriding safety concerns with regard to the use of recombinant viral based vectors in the genetic manipulation of stem cells.78 It remains uncertain as to whether legislation would ultimately permit the use of genetically modified stem cells for human clinical therapy. At present, the potential detrimental effects of transplanting genetically modified stem cells in vivo are not well studied. More research needs to be carried out on animal models to address the safety aspects of such an approach.

More recently, there is emerging evidence that some transcription factors (which are commonly thought of as cytosolic proteins) have the ability to function as paracrine cell to cell signaling molecules.79 This is based on intercellular transfer of transcription factors through atypical secretion and internalization pathways.79 Hence, there is an exciting possibility that transcription factors implicated in commitment to the keratinocyte lineage may in the future be genetically engineered to incorporate domains that enable them to participate in novel paracrine signaling mechanisms. This in turn would have tremendous potential for inducing the commitment of ES cells and non-epidermal adult stem cells to the keratinocyte lineage.

Skin appendages, including hair follicles, sebaceous glands and sweat glands, are linked to the epidermis but project deep into the dermal layer. The skin epidermis and its appendages provide a protective barrier that is impermeable to harmful microbes and also prevents dehydration. To perform their functions while being confronted with the physicochemical traumas of the environment, these tissues undergo continual rejuvenation through homeostasis, and, in addition, they must be primed to undergo wound repair in response to injury. The skins elixir for maintaining tissue homeostasis, regenerating hair, and repairing the epidermis after injury is its stem cells.

The hair follicle is composed of an outer root sheath that is contiguous with the epidermis, an inner root sheath and the hair shaft. The matrix surrounding the dermal papilla, in the hair root, contains actively dividing, relatively undifferentiated cells and is therefore a pocket of MSCs that are essential for follicle formation. The lower segment of each hair follicle cycles through periods of active growth (anagen), destruction (catagen) and quiescence (telogen).80 A specialized region of the outer root sheath of the hair follicle, known as the bulge, is located below the sebaceous gland, which is also the attachment site of the arrector pili muscle, receiving inputs from sensory nerve endings and blood vessels. Furthermore, the hair follicle bulge is a reservoir of slow-cycling multipotent stem cells.81, 82 Subsets of these follicle-derived multipotent stem cells can be activated and migrate out of hair follicles to the site of a wound to repair the damaged epithelium; however, they contribute little to the intact epidermis. These hair follicle stem cells can also contribute to the growth of follicles themselves and the sebaceous gland. For example, in the absence of hair follicle stem cells, hair follicle and sebaceous gland morphogenesis is blocked, and epidermal wound repair is compromised.83 In addition to containing follicle epidermal stem cells, the bulge contains melanocyte stem cells.84 Recent studies show that nestin, a marker for neural progenitor cells, is selectively expressed in cells of the hair follicle bulge and that these stem cells can differentiate into neurons,85 glia, keratinocytes, smooth muscle cells, melanocytes and even blood vessels.86, 87 Examination of close developmental and anatomical parallels between epithelial tissue and dermal tissue in skin and hair follicles has revealed dermal tissue to have stem cells. Paus et al. indicated that hair follicle dermal sheath cells might represent a source of dermal stem cells that not only incorporate into the hair-supporting papilla, low down in the follicle, but also move up and out from the follicle dermal sheath into the dermis of adjoining skin.88 Hair follicle dermal sheath cells taken from the human scalp can form new dermal papilla, induce the formation of hair follicles, and produce hair shafts when transplanted onto skin.89 There is also a clear transition from dermal sheath to dermal papilla cells.90 When the follicle dermal cells are implanted into skin wounds, they can be incorporated into the new dermis in a manner similar to that of skin wound-healing fibroblasts.91 However, these cell populations still lack specific markers for purifying and distinguishing the stem cells from their progeny. Furthermore, of prime importance is improving our understanding of the relation between bulge cells and interfollicular epidermal stem cells and between bulge cells and other stem cells inhabiting the skin and the mechanisms of hair growth.

Recently, cell replacement therapy has offered a novel and powerful medical technology for skin repair and regeneration: a new population of stem cell, called a neural crest stem cell, from adult hair follicles, was discovered to have the ability to differentiate in vitro to keratinocytes, neurons, cartilage/bone cells, smooth muscle cells, melanocytes, glial cells, and adipocytes.9296 In mammalian skin, skin-derived neural progenitors were isolated and expanded from the dermis of rodent skin and adult human scalp and could differentiate into both neural and mesodermal progeny.97, 98 Skin-derived neural progenitor cells were isolated based on the sphere formation of floating cells after 37 days of culture in uncoated flasks with epidermal growth factor and fibroblast growth factor, and characterized by the production of nestin and fibronectin, markers of neural precursors. In addition, skin-derived neural progenitor cells were identified as neural crest derived by the use of Wnt1 promoter driving LacZ expression in the mouse. Some of the LacZ-positive cells were found in the skin of the face, as well as in the dermis and dermal papilla of murine whisker.99 These skin derived neural crest cells have already shown promising results in regenerative medicine such as the promotion of regenerative axonal growth after transplantation into injured adult mouse sciatic nerves 95 or spinal cord repair,100 resulting in the recovery of peripheral nerve function. This new study marks an important first step in the development of real stem-cell-based therapies and skin tissue regeneration.

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Stem Cells for Skin Tissue Engineering and Wound Healing

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genucel - Intensive New Stem Cell Eye Therapy Treatment ...

Vista, Ca, (PRWEB) November 23, 2014

DC Labs, nationwide provider of the luxurious Ovation Cell Therapy, introduces new retail opportunity to their Salon Professional Division. Previously, the Ovation Hair product line was available exclusively online.

Ovation Hair launched their Salon Professional Division in 2013, to offer training and certification to licensed stylists interested in learning about the benefits of Cell Therapy as well as offer In-Salon treatments to their clients. The program was received with huge success and has grown exponentially since its inception.

Lead Stylist, Brenda Stearns explains, As Certified Cell Therapists, we have been requesting this retail opportunity with Ovation since we began offering In-Salon Cell Therapy Treatments. Based on years of salon experience, we were certain clients would want to walk out of the salon product in hand - once we introduced the amazing benefits of thicker, stronger, longer hair to them. Ms. Stearns continues, This is truly a fabulous opportunity for us as stylists as well as for our clientele.

Ovation Hair is releasing the entire line of hair care products to salons currently offering Cell Therapy Treatments. The complete Ovation hair care line includes shampoos, conditioners and styling products that address individual hair care needs based on hair condition and type.

Dallas Van Kempen, CEO at Ovation Hair and founder of the Salon Professional Division explains, When we initially launched this In-Salon program, I approached it as a unique inventory free opportunity. Allowing salon stylists to simply direct interested clients to the Ovation Hair website for purchasing product. I wanted to take the burden out of carrying inventory and stocking shelves. What we came to realize was that the salon client does not want to wait for their product to be shipped to them. Mr. Van Kempen continues, Now they dont have to. The option for non-inventory support will continue to be available.

Ovation expects in-salon retail offerings to grow as more salons nationwide provide Cell Therapy Treatments. To become a Certified Cell Therapist and offer Cell Therapy Treatments and retail product at your salon, please visit http://www.ovationpro.com.

About Ovation Hair The philosophy of Ovation Hair is to meet clients high expectations with quality, nourishing and rejuvenating products that demonstrate proven effectiveness. Ovation Hair has been helping clients achieve healthy hair since 2007. Their flagship product, Ovation Cell Therapy, is proven to deliver thicker, stronger, longer hair. Ovation products include high quality ingredients, demonstrated to be safe and effective to create healthy hair. The hair care products are developed as a natural alternative designed to rejuvenate hair to look thicker, stronger and longer. For more information visit http://www.ovationhair.com. ###

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Ovation Hair Introduces Retail Product to Salons

Catholic Edge Genetic Engineering HD
Video By: Austin Riordan.

By: Anna Landis

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Catholic Edge Genetic Engineering HD - Video

Might a GMO potato really be a better potato than can win over opponents of genetic engineering and feed the French fry minions? Image: J.R. Simply.

J.R. Simplot won federal approval for a genetically modified potato on Nov. 7, and although it is a major supplier of french fries to McDonald's , don't worry about some GMO spud showing up in your Happy Meal. The fast-food chain says it has no plans on adopting it anytime soon.

But that might not be the case for long.

Simplot says that because its new potato is made from genes derived from potatoes themselves and not bacteria or genes from other species, thus creating some sort of Frankenfood, it doesn't rise to the same level of concern activists would have over some crops, or when they thwarted Monsanto's effort to propagate a GMO potato in the 1990's.

Back then, Monsanto got the FDA to approve its New Leaf potatoes that were resistant to disease and insects through the development of a synthetic version of the bacillus thuringiensis bacterium, or Bt.

After initial acclaim, the biotech charged high premiums for its seed, which limited adoption, and then ultimately went nowhere when McDonald's and the Frito-Lay division of PepsiCo were cowed by anti-GMO activists into rejecting them. J.R. Simplot, an early adopter of the GMO potato, instructed its farmers not to plant them. Monsanto eventually stopped developing the potato in 2001.

Simplot's new Innate potatoes was developed to be especially beneficial to fast-food restaurants that sell lots of french fries. It's a little science-y, but it's important to understanding what Simplot is doing.

When potatoes and other starchy foods like bread (and even coffee!) are processed at high temperatures, such as when they're deep-fried, a carcinogenic chemical compound called acrylamide if formed.

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A GMO Potato Is Approved, But Will Anyone Buy It?

The Blockheads Pro Tips SERIES 2: Tulip Genetics!
To start off the new series of Pro Tips, I show you how to use Tulip Genetics to make your own Tulip hybrids! SUBSCRIBE FOR A HIGH FIVE! http://bit.ly/1fPJDRC (/._.)/*(._.) JOIN SAM.BOYER...

By: Sam.Boyer

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The Blockheads Pro Tips SERIES 2: Tulip Genetics! - Video

Las Vegas, NV (PRWEB) November 24, 2014

Exclusive Genetics, a leader in the bucking bull industry returns to Las Vegas, Nevada for the fourth consecutive year to feature its $1.5 million Bucking Bull Games. The Games will be held December 4-13 parallel to the Wrangler National Finals Rodeo, when over 250,000 fans converge on Las Vegas for the world series of rodeo.

Bucking Bull Games will present, over the course of 10 days, five bucking bull events with a record high $1.5 million in payouts to winners. Championships will be determined in categories including the Million Dollar Bucking Bull Championship Final Round, the Second Chance Championship, and the Finale of the Bull and Rider Tournament.

This prestigious event will also feature a special event called Buck Autism Weekend. Sponsored by Branded Cities Network, the campaign aims to raise funds and awareness to support families who have children diagnosed with autism.

Billy Jaynes, CEO of Orchard, Texas-based Exclusive Genetics, Bucking Bull Games and the Million Dollar Bucking Bull Championship, as well as Founder of Buck Autism is confident that, Folks in the bucking bull community are some of the most generous and compassionate people I know. They rally around a cause and I am happy that Buck Autism and its goals have touched so many people in a positive way. He added, We are also pleased to see corporations supporting the campaign, including Branded Cities Network. Their displaying of a Public Service Announcement on the Harmon Corner Digital Board in Las Vegas will be paramount to raising awareness about the events in support of the National Autism Association.

Wendy Fournier of the National Autism Association expressed her gratitude in being able to be involved with the Buck Autism Campaign saying, "With 1 in 68 children in the United States affected by autism, we are blessed to have partnered with Billy Jaynes who continues to work hard to raise awareness of NAA within the bucking bull industry. We are looking forward to the December Bucking Bull Games Championship where we will continue the Campaign including charity events encompassing Buck Autism Weekend.

Events will be held both at Cowboy Marketplace in the Mandalay Bay Hotel and Casino, as well as the historic rodeo arena at Horsemans Park next to Sams Town Hotel and Gambling Hall. Bucking Bull Games is sponsored by Purina Animal Nutrition LLC and Sams Town Hotel & Gambling Hall.

For more information and the complete event schedule visit http://www.BuckingBullGames.com. To support the Buck Autism campaign visit http://www.BuckAutism.com.

About Bucking Bull Games Exclusive Genetics Bucking Bull Games presents six major events during the Wrangler National Finals Rodeo in Las Vegas, NV that offer industry leading payouts of over $1,500,000. The competitions include the two-year-old Million Dollar Bucking Bull Championship and Second Chance Championship, yearling Gold Buckle Challenge and Million Dollar Bucking Bull and Rider Tournament for three-, four- and five-year-olds. Visit http://www.exclusivegenetics.com/bucking_bull_games.

About Buck Autism "Buck Autism founded by Billy Jaynes, CEO of Exclusive Genetics, stems from how one persons life changed dramatically after getting involved in the bucking bull world. Wyoming resident, Jayci Mead, a 21-year-old woman who has autism, is involved in bull ownership through Exclusive Genetics. Her interest in bucking bulls has helped her communicate and express herself in ways that she was unable to do so prior to her connection to bull ownership. To learn more or to see Jaycis complete story and video at http://www.BuckAutism.com.

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Exclusive Genetics Bucking Bull Games to Highlight Buck Autism Weekend and Charity Fundraiser in December in Support ...

Stem Cell Cure for "Bubble Baby" Disease (SCID), Pioneered by UCLA #39;s Don Kohn
On November 18th, 2014, a UCLA research team led by Donald Kohn, M.D., announced a breakthrough gene therapy and stem cell cure for "bubble baby" disease, or severe combined ...

By: California Institute for Regenerative Medicine

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Stem Cell Cure for "Bubble Baby" Disease (SCID), Pioneered by UCLA's Don Kohn - Video

preservation laboratory, where stem cells were kept for use in transplants in children whose bone marrow has been damaged during chemotherapy.

Concerns were first raised when Sophie Ryan-Palmer, 12, who had acute lymphoblastic leukaemia, failed to make progress after her transplant in June 2013, which involved using a donors stem cells rather than her own.

She had been diagnosed with leukaemia at the age of two and had undergone three previous transplants. She began fundraising for cancer charities when she was six.

By October last year the hospital had identified that a higher than usual proportion of eight children who had undergone stem cell transplantation between March and August had suffered what doctors call delayed engraftment. But by the time it stopped freezing stem cells on site at its base in Bloomsbury, central London, and launched an investigation, three of the eight had died.

Ryan Loughran, aged 13 months, from Bournemouth, died on July 10. Sophie, from Sunbury in Middlesex, followed on July 17. Katie Joyce, from Hertfordshire, died on October 6. A fourth patient, Muhanna al-Hayany, aged five, died in August this year. He had come from Kuwait to have the treatment. Following the deaths it was discovered that the method used to freeze the stem cells had inexplicably stopped working and that, although still alive, the cells were unable to mature properly.

At the inquest, Katie Beattie, the barrister representing Katie Joyces family, questioned whether the girls transplant in August should have been suspended, knowing Sophie and Ryan had died the previous month. Great Ormond Street went ahead even though there was plenty of time to stop it, she said.

Doctors from the hospital told St Pancras coroners court that they regretted not halting transplants sooner and Katies life might have been saved if they had. But they said they believed they were doing the right thing by continuing with the transplants because cancer doesnt wait.

Great Ormond Street said it has since overhauled its procedures to prevent further incidents, but is still investigating why the freezing process stopped working.

A spokesman said: Before giving our patients any frozen cells we carried out tests, which are standard across most laboratories in the UK, to ensure they were alive and viable. All of the samples passed these tests, so there was nothing to suggest there was a problem at this stage.

The coroner, Mary Hassell, is expected to deliver verdicts on all four deaths on Tuesday.

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Stem cell failure 'led to children's deaths' at Great Ormond Street

Patients, aged one to 12, among eight children whose transplants failed Concerns arose in 2013 after operation on fundraiser Sophie Palmer, 12 Hospital says Katie Joyce, 4, could have been saved if quicker action taken Lawyers have also accused hospital of taking too long to stop transplants Doctors 'regret' not stopping sooner but decision seemed right at time Ryan Loughran, 13 months, and Muhanna al-Hayany, 4, also died last year Seventeen months on, investigations are still ongoing into exact cause

By Steph Cockroft for MailOnline

Published: 06:45 EST, 22 November 2014 | Updated: 12:57 EST, 22 November 2014

Four cancer-stricken children died at Great Ormond Street Hospital after a series of failures in stem cell transplants at the world-renowned hospital, an inquest has heard.

The young patients, aged between one and 12, were among eight children whose transplants failed when the stem cell freezing system - used in life-saving operations - inexplicably stopped working.

Four children went on to recover. But well-known charity fundraiser Sophie Ryan Palmer, 12, one-year-old Ryan Loughran, four-year-old Katie Joyce and Muhanna al-Hayany, also four, died between July and October last year.

Katie Joyce (left) and Sophie Ryan (right) were among two of the four young patients who died after a series of failures in stem cell transplants at Great Ormond Street Hospital

The children's hospital has now admitted that Katie might have survived if it had acted more quickly to resolve the problems.

Lawyers for two of the families have also accused Great Ormond Street of taking too long to stop the transplants once concerns arose.

At an inquest into the deaths this week, the court heard that doctors were initially dumbfounded as to why the procedures suddenly started failing after a decade of success, the Guardian reports.

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Four children dead at Great Ormond Street after stem cell transplant failure

The skin is the body's first line of defense against infection. And when this barrier is broken, or an internal organ is ruptured, it is the process of coagulation, or clotting, which relies largely on blood cells called platelets, that seals the breach and stems the flow of blood. Researchers at UC Santa Barbara (UCSB) have now synthesized nanoparticles that mimic the form and function of platelets, but can do more than just accelerate the body's natural healing processes.

Platelets are nucleus-free blood cells that are essentially the building blocks for any blood clot, binding together at the edge of a wound as well as changing shape and secreting chemical messengers to call more platelets to the scene of the injury to assist in the healing process. However, coagulation can be hampered if an injury is too severe, or if the injured person is taking anti-coagulation medication or suffers from a platelet disorder.

In such cases, the platelet-like nanoparticles (PLNs) synthesized at UCSB could save the day. Developed by researchers in UCSB's Department of Chemical Engineering and its Center for Bioengineering, the synthetic platelets behave just like their naturally-occurring counterparts, mimicking their shape, flexibility and surface biology.

In this way, their creators say they could be added to a patient's own natural blood supply or augment the patient's own platelet supply and accelerate the healing process for both internal and external injuries. Not only will the PLNs congregate at the site of an injury, but like natural platelets, they'll also call other platelets to the site and bind to them. Then, once their task has been completed and the wound has been plugged, the PLNs will dissolve into the blood.

Furthermore, the researchers claim to have improved on nature, with their nanoscale synthetic platelets outperforming micron-sized natural ones in tests. The synthetic platelets are also able to be customized with medications that match the needs of patients with a specific condition. They could also be used to carry antibiotics to certain parts of the body, such as across the blood-brain barrier, to provide targeted therapy and improved diagnostics.

"This technology could address a plethora of clinical challenges," said Dr. Scott Hammond, director of UCSBs Translational Medicine Research Laboratories. "One of the biggest challenges in clinical medicine right now which also costs a lot of money is that were living longer and people are more likely to end up on blood thinners." The researchers say PLNs would help in the treatment of such elderly patients, allowing the PLNs to be targeted at the site of an injury without triggering unwanted bleeding.

Importantly, the researchers say the synthetic platelets have a longer shelf life and are cheaper relatively speaking than another person's platelets both important factors at times of emergencies and natural disasters when blood products are in highest demand.

These aren't the first synthetic platelets weve seen that improve on nature. In 2009, researchers at Case Western University reported the development of artificial platelets made of biodegradable polymers that reduced clotting times in animal models and attracted the interest of the military.

The team will now look at how well the production of the PLNs scales up and asses practical clinical concerns, such as manufacturing, storage, sterility ahead of pre-clinical and clinical testing.

Results of the researchers current findings appear in the journal ACS Nano.

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Platelet-like nanoparticles improve on nature to stem the blood flow

Stem Cell Therapy: Dr. Roberta Shapiro - A NY Physician #39;s Path to Panama
Special Guest Speaker, Roberta F. Shapiro DO, FAAPM R speaks about: A New York Doctor #39;s Path to Panama at the Stem Cell Institute #39;s Stem Cell Therapy Publi...

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Stem Cell Therapy: Dr. Roberta Shapiro - A NY Physician's Path to Panama - Video

Buddy the beagle can walk again
Buddy the beagle wasn #39;t able to walk when he first arrived at the University of Minnesota Veterinary Medical Center. With the help of U of M veterinarians and staff, using stem-cell therapy,...

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Buddy the beagle can walk again - Video

Genetic Engineering Mash Up

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Genetic Engineering Mash Up - Video

Mower Genetics - itn iek

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Mower Genetics - itn iek - Video

THANK YOU from Saving Eliza!
THANK YOU to all the supporters of Saving Eliza and helping make history! YOU did it! You #39;ve made a significant impact on stopping Sanfilippo Syndrome for this generation and future generations!...

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HOPE, HYPE REALITY: ZINC FINGER GENE THERAPY TO CONTROL OR CURE HIV
A video to answer your questions about a gene therapy approach to an HIV cure. On Nov 5, 2014, the defeatHIV Community Advisory Board hosted a talk with Dr. Philip D. Gregory (Chief Scientific...

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HOPE, HYPE & REALITY: ZINC FINGER GENE THERAPY TO CONTROL OR CURE HIV - Video

By Cyndi Root

The Vaccine & Gene Therapy Institute of Florida (VGTI), a non-profit research institute, and TapImmune have formed a partnership to develop TapImmunes cancer vaccines. The companies announced the collaboration in a press release stating that they will move experimental vaccines for breast and ovarian cancers into Phase 2 clinical trials.

Keith Knutson, PhD, VGTIs Director of Cancer Vaccines and Immune Therapies Program, explained the need for vaccines, All it takes is a few malignant cells to continue to circulate in the body until they eventually anchor and metastasize. Because these cancer cells already survived primary therapy, they are typically drug-resistant and much more difficult to treat.

VGTI and TapImmune Agreement

VGTI and TapImmune have agreed to coordinate efforts on cancer vaccines, including study design and trial site selection. VGTI will work with TapImmune to recruit clinical advisors, select manufacturers, and procure outsourced resources as necessary. The two will also work together in executing the clinical trials. Upon successful regulatory approvals, TapImmune holds the exclusive commercialization rights for the vaccines.

Cancer Vaccine Candidates

Investigators from VGTI and TapImmune hope to vaccinate women who have achieved remission in their breast or ovarian cancer in order to prevent cancer recurrence. Dr. Knutson said that cancer survivors have a substantial rate of cancer returning due to malignant cells that escaped during primary treatment. Antigens, determined by genetic and molecular profiling, in the vaccine would work to target the proteins expressed on the patients tumor cells, triggering an immune response with few side effects. The immune system would eliminate rebel cancer cells and stop new ones from growing.

Cancer Vaccines

Cancer vaccines are being engineered to boost the immune system, kickstarting it so it will kill abnormal cells and prevent malignant cell growth. Cancer vaccines are distinguished according to prevention or treatment. The Food and Drug Administration (FDA) has approved prevention vaccines for the hepatitis B virus, which can cause liver cancer, and human papillomavirus (HPV). Clinical trials for treatment vaccines are much more numerous than those for preventative vaccines. The National Cancer Institute (NCI) is currently listing 12 trials for vaccines to prevent cervical cancer and three to prevent solid tumors.

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Vaccine & Gene Therapy Institute, TapImmune Partner On Cancer Vaccines

Roughly 40 million people across the world are blind and, for a long time, most forms of blindness were permanent conditions. The same situation held for degenerative diseases that affect eyesight.

But recently, scientists have made some surprising headway into changing that. New treatments like gene therapy, stem-cell therapy, and even bionic implants are already starting to restore some patients' sight. And these technologies are expected to keep improving in the future.

Here's a look at all the ways scientists have tried and, increasingly, succeeded in curing the blind:

Children's Hospital of Philadelphia, Daniel Burke/AP Photo This undated image released by the Children's Hospital of Philadelphia shows doctors Albert Maguire, left, along with wife Jean Bennett at the University of Pennsylvania. The two are part of two teams of scientists in the United States and Britain that are using gene therapy to dramatically improve vision in four patients with an inherited eye disease that causes blindness in children.

Tweaking genes is one promising route to treat blindness.

In 2011, a group led by Jean Bennett of the University of Pennsylvania used gene therapy to treat some patients with a congenital blindness disorder. The patients in question all hada hereditary disease called Leber congenital amaurosis, and they all had mutations in their RPE65 gene.The patients were each given a non-harmful virus that could sneak a healthy copy of the gene into their eye cells. Six out of 12 showed improvement.

Then, in 2014, researchers led by Robert MacLaren, an ophthalmologist at Oxford,presented some promising early results of a very smallstudy of six patients at various stages of a rare, inherited disease calledchoroideremia. These patients all lacked a protein calledREP1, which leads to progressive vision loss. Doctors took the gene forREP1, put it in a non-harmful virus, and injected that virus into the patients' eyes. All reported some improvement in their sight.

"One patient, who before his treatment could not read any lines on an eye chart with his most affected eye, was able to read three lines with that eye following his treatment,"wrote Susan Young Rojahn at MIT Technology Review.

Commercial treatments are still a ways off, however. Researchers first have to continue to monitor these patients to see what happens to their vision over the long term (and check for side effects).The FDA currently recommends 15 years of safety monitoring before trying to get a specific gene therapy approved.

2) Stem cells

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4 strategies doctors are using to cure the blind

2nd International Conference onPredictive Preventive Personalized Medicine Molecular Diagnostics
2nd International Conference on Predictive, Preventive and Personalized Medicine Molecular Diagnostics will be organized during November 3-5, 2014, at Embassy Suites Las Vegas, USA with the...

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AVON LAKE, OH (WOIO) - When Shannon Goulding's bloodhound Butler tore a ligament in his knee his entire personality changed.

"He was sedentary, and he wasn't as active as before," said Goulding.

Dr. Petti a veterinarianat the Avon Lake Animal Clinic told Goulding, who also works at the clinic, suggested that stem cell therapy could help.

"Watching him walk he looked stiff and uncomfortable," said Petti.

The therapy was successful. Goulding said after four weeks after the surgery she could see a change the way Butler moved.

Stem cell therapy helps animals suffering from sore knees and joints by using their own fat cells.

"You take them from the patient, you process them, make them active, and then you re inject them into the parts of the animal that are giving them problems," said Petti.

Petti said Avon Lake Animal Clinic has helped about 15 animals with stem cell therapy and people from all over the country have been calling.

One injection of stem cells can last up to three years, and after that a second injection may be needed.

Stem cell therapy is also an expensive procedure. It ranges from $2,000-2,500, but for Goulding she says seeing Butler run free without pain is worth it.

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Local clinic treats animals with stem cell therapy

Researchers of CEU Cardenal Herrera University (CEU-UCH) for the first time transplanted bone marrow stem cells into damaged brain tissue while applying lipoic acid (a potent antioxidant), with the aim of improving neuroregeneration in the tissue. This new way of repairing brain damage, which combines cellular treatment with drug therapy, has shown positive results, especially in forming blood vessels (a process called angiogenesis) in damaged areas of the brains of adult laboratory mice. Angiogenesis is a process that is essential to the recovery of damaged neural tissues. The investigation was led by Jos Miguel Soria Lpez, deputy director of the Institute of Biomedical Sciences at CEU-UCH, and its results were published in the international medical journal Brain Injury.

Professor Soria, who is affiliated to the Department of Biomedical Sciences at CEU-UCH, heads the investigative group 'Strategies in Neuroprotection and Neuroreparation', which carried out the investigation in cooperation with the Andalusian Molecular Biology and Regenerative Medicine Centre (CABIMER), located in Sevilla, and the Mediterranean Ophthalmological Foundation, located in Valencia. The research team used the experience they obtained from their previous investigations on the neuroregenerative efficiency of lipoic acid to develop a new remediation strategy for patients of brain damage. This new therapy combines the transplantation of bone marrow stem cells into the brain -- in this case, the brains of adult rats -- with the administration of the potent antioxidant lipoic acid.

Lipoic acid is already used in the treatment of degenerative diseases such as multiple sclerosis or diabetic neuropathy. Professor Soria concluded from previous researches he conducted at CEU-UCH that it has the ability to increase the creation of blood vessels, which speeds up cerebral immune response after an injury and stimulates the restoration of damaged tissues. Several other researches, for their part, proved that after brain damage stem cell therapies using a patient's own bone marrow induce functional improvements. The two therapies -- one cellular; the other one pharmacological -- were both applied in this research so as to evaluate their combined effect.

New blood vessels

Angiogenesis -- the process that forms new blood vessels -- in the treated neuronal tissue began only eight days after the application of this new, combined therapy. CEU-UCH professor Soria says that "although bone marrow stem cells disappear from the brain tissue where they were transplanted after only 16 days, new cells keep forming. To put it another way, brain tissue is regenerated by new cells that appear in the brain as a result of stem cell transplantation. This proves the regenerative efficiency of the new combined therapy."

The research also shows how the blood vessels that formed after the treatment grow into the damaged area of the brain. "They act as a kind of scaffolding to that area that allows microglia cells to migrate," professor Soria says. "In the damaged area, they contribute to regeneration." He adds that "the application of both treatments results into high angiogenic activity, which is crucial for an efficient recovery of the damaged brain area." According to Soria, "the laboratory mice that recovered fastest from brain injuries were those that had a higher density of regenerated blood vessels."

Taking into consideration brain damage is, especially among children and adolescents, one of the leading causes of disability and death in the developed world, the good results that were obtained from the combination of the two therapies make the research team very hopeful. "Combining an antioxidant such as lipoic acid with bone marrow stem cells has proven to be an effective remedy," Soria observes. The team plans to conduct future research into similar combined therapies.

The image above shows the transplant of bone marrow stem cells from transgenic mice under the effects of cerebral cortex after suffering local brain damage. Also visible is a neuroprotective drug therapy.

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Brain injuries in mice treated using bone marrow stem cells, antioxidants