For breast cancer patients, the era of personalized medicine may be just around the corner, thanks to recent advances by USC Stem Cell researcher Min Yu and scientists at Massachusetts General Hospital and Harvard Medical School.

In a July 11 study in Science, Yu and her colleagues report how they isolated breast cancer cells circulating through the blood streams of six patients. Some of these deadly cancer cells are the "seeds" of metastasis, which travel to and establish secondary tumors in vital organs such as the bone, lungs, liver and brain.

Yu and her colleagues managed to expand this small number of cancer cells in the laboratory over a period of more than six months, enabling the identification of new mutations and the evaluation of drug susceptibility.

If perfected, this technique could eventually allow doctors to do the same: use cancer cells isolated from patients' blood to monitor the progression of their diseases, pre-test drugs and personalize treatment plans accordingly.

In the six estrogen receptor-positive breast cancer patients in the study, the scientists found newly acquired mutations in the estrogen receptor gene (ESR1), PIK3CA gene and fibroblast growth factor receptor gene (FGFR2), among others. They then tested either alone or in combination several anticancer drugs that might target tumor cells with these mutations and identified which ones merit further study. In particular, the drug Ganetspib -- also known as STA-9090 -- appeared to be effective in killing tumor cells with the ESR1 mutation.

"Metastasis is the leading cause of cancer-related death," said Yu, assistant professor in the Department of Stem Cell Biology and Regenerative Medicine at the Keck School of Medicine of USC. "By understanding the unique biology of each individual patient's cancer, we can develop targeted drug therapies to slow or even stop their diseases in their tracks."

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The above story is based on materials provided by University of Southern California - Health Sciences. The original article was written by Cristy Lytal. Note: Materials may be edited for content and length.

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Im not betting on it, but if, by some miracle, Argentine winger Angel Di Maria is on the pitch against Germany Sunday in the 2014 World Cup final, get ready for another explosion of interest in stem cell therapy, a now familiar occurrence every time a famous athlete undergoes the treatment.

Di Maria, who either tore or strained a thigh muscle in Argentinas World Cup win over Belgium, is so determined to play in the final that, according to some reports, he is having the muscle injected with stem cells in the hope of healing by Sunday. (This Associated Press report from Thursday said he was practicing at 60 to 80 percent, so Im guessing were talking about a strain.)

If those reports are true, Di Maria will join a long line of elite athletes who have resorted to the unproven and possibly risky therapy. This kind of stem cell therapy is experimental in every sense of the word, according to the International Society for Stem Cell Research. There also is some evidence that the procedure can promote tumor growth or create an immune response to a patients own cells, or that injected stem cells might migrate to another part of the body.

Never mind. There is soccer to be played!

No one denies that stem cells hold promise as a therapy down the road, perhaps in as little as five or 10 years, says Kevin McCormack, communication director for the California Institute for Regenerative Medicine. With $3 billion supplied by voters in a 2004 ballot initiative, the organization is funding trials of the use of stem cell therapies for scarring after heart attacks, sickle cell anemia, leukemia and other conditions.

But for now, stem cells are known to be effective only for certain disorders of the blood, immune system and bone marrow. Beyond that, little has been proven, although clinics in the United States and around the world are offering the therapy and raking in bucks from desperate patients.

Di Maria may even see some benefits, McCormack said. In theory, they might [help] because they may have an anti-inflammatory effect or they may stimulate the bodys own natural healing, he said. But the problem is that they havent done any research to prove that.

The stem cells are harvested from a patients bone marrow and sometimes run through a centrifuge to concentrate them. Then they are injected into the damaged tissue.

For athletes, who are always looking for ways to prolong their careers and bounce back from injury, the fad began in 2010, when Major League pitcher Bartolo Colon had a slurry of stem cells that can turn into a variety of tissues injected into his injured elbow and shoulder. Within months, he was throwing 93 mile per hour fastballs for the New York Yankees. Later, Denver Broncos quarterback Peyton Manning, one of the most famous athletes in the United States, reportedly had stem cell therapy on his injured neck.

McCormack and others express concern that when pro athletes and other celebrities have unproven treatments, it sends the rest of us weekend warriors out in search of the same. Here a good bit of blame goes to us in the media. A 2012 analysis conducted for the journal Molecular Therapyshowed that 72.7 percent of the media coverage of athletes and stem cell therapy didnt address whether the treatment works, and 42 percent referred to alleged benefits. Only 5.7 percent of the stories brought up possible safety issues and risks.

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11-Jul-2014

Contact: Jia Liu liujia@genomics.cn BGI Shenzhen

July 3, 2014, Shenzhen, China Researchers from Salk Institute for Biological Studies, BGI, and other institutes for the first time evaluated the safety and reliability of the existing targeted gene correction technologies, and successfully developed a new method, TALEN-HDAdV, which could significantly increased gene-correction efficiency in human induced pluripotent stem cell (hiPSC). This study published online in Cell Stell Cell provides an important theoretical foundation for stem cell-based gene therapy.

The combination of stem cells and targeted genome editing technology provides a powerful tool to model human diseases and develop potential cell replacement therapy. Although the utility of genome editing has been extensively documented, but the impact of these technologies on mutational load at the whole-genome level remains unclear.

In the study, researchers performed whole-genome sequencing to evaluate the mutational load at single-base resolution in individual gene-corrected hiPSC clones in three different disease models, including Hutchinson-Gilford progeria syndrome (HGPS), sickle cell disease (SCD), and Parkinson's disease (PD).

They evaluated the efficiencies of gene-targeting and gene-correction at the haemoglobin gene HBB locus with TALEN, HDAdV, CRISPR/CAS9 nuclease, and found the TALENs, HDAdVs and CRISPR/CAS9 mediated gene-correction methods have a similar efficiency at the gene HBB locus. In addition, the results of deep whole-genome sequencing indicated that TALEN and HDAdV could keep the patient's genome integrated at a maximum level, proving the safety and reliability of these methods.

Through integrating the advantages of TALEN- and HDAdV-mediated genome editing, researchers developed a new TALEN-HDAdV hybrid vector (talHDAdV), which can significantly increase the gene-correction efficiency in hiPSCs. Almost all the genetic mutations at the gene HBB locus can be detected by telHDAdV, which allows this new developed technology can be applied into the gene repair of different kinds of hemoglobin diseases such as SCD and Thalassemia.

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Stem cells grown under low oxygen. These stem cells from Stemedica are licensed to CardioCell.

CardioCell, a San Diego stem cell company, has started a Phase 2a trial of its treatment for chronic heart failure.

The companys special stem cells will be injected into patients with heart failure not caused by a heart attack. Nearly 2 million Americans have that kind of heart failure.

CardioCell is also testing these stem cells on heart attack patients to help their recovery. The cells are licensed from Stemedica, CardioCell's parent company.

Taken from bone marrow, the stem cells produce chemicals intended to heal malfunctioning heart cells. They are grown under low oxygen conditions, or hypoxia. CardioCell says hypoxia reflects the conditions under which natural stem cells exist. Histogen, also of San Diego, is developing its own kind of low-oxygen stem cells.

Growing stem cells with abundant oxygen reduces their "stemness," and they become prone to differentiate, said Sergey Sikora, CardioCell's president and chief executive.

Sergey Sikora, president and CEO of CardioCell / CardioCell

More than 20 patients are being sought to take part in the study, which is taking place at three locations. These are Emory University in Atlanta, Northwestern University in Chicago, and the University of Pennsylvania in Philadelphia.

Patients will receive injections of the stem cells, and a control group will receive a saline injection. After 90 days, the groups will be reversed. Patients who had received the stem cells will get a saline injection, and the control group will get the stem cells.

The stem cells last for about a month, after which they disappear, Sikora said.

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Tampa, FL (PRWEB) July 10, 2014

The Lung Institute works to help people who have received a diagnosis of debilitating lung disease get their quality of life back. The latest case study demonstrates how stem cell therapy can be used effectively to treat interstitial lung disease. After his recent stem cell treatment at the Lung Institute, Al Corter can now complete his daily tasks on his horse farm much faster, and finally attend the Silver Spur Riding Club Open Horse Show the weekend of July 12th in Fonda, NY.

Twelve years ago, Al was exposed to toxic fumes in the workplace and subsequently diagnosed with interstitial lung disease and bronchiectasis, a form of chronic obstructive pulmonary disorder (COPD). Living in upstate New York and running his horse farm, Als serious pulmonary conditions had a major effect on his life. Shortness of breath, coughing, reliance on supplemental oxygen and fatigue were taking a toll. Al needed a new solution.

Al decided to travel to Florida to undergo stem cell treatment at the Lung Institute facility in Tampa. He was seeking an alternative treatment to help with his symptoms. Stem cell therapy is a minimally invasive process that involves extracting stem cells, and then reintroducing them to cue the bodys natural healing processes. The stem cells are taken from the patients own body, so there is no controversy or risk of rejection.

Stem cell therapy is a viable option for many people with lung disease, said Dr. Burton Feinerman, Medical Director of the Lung Institute. Our patients are breathing easier, walking further and depending less on supplemental oxygen.

Prior to stem cell therapy, Al was needing more and more supplemental oxygen. His quality of life had taken a sharp turn downward. Following adipose stem cell treatment, Al is feeling better. He is getting back to the routine at the farm. Al used to use 5 to 6 liters of continuous oxygen to get his outdoor farm work done. Now, he is able to do these daily chores faster, and uses only 4 to 5 liters of oxygen on a pulsing regulator.

Im getting everyday tasks done quicker, said Al. Im using about half the amount of oxygen as I was before to do the same activities. My quality of life has definitely improved.

The Lung Institute has treated hundreds of patients with lung disease from around the country and the world. Regardless of the stage of the disease, patients are able to undergo stem cell therapy, which helps damaged lung tissue, and can lessen their symptoms.

About Lung Institute At the Lung Institute, we are changing the lives of hundreds of people across the nation through the innovative technology of regenerative medicine. We are committed to providing patients a more effective way to address pulmonary conditions and improve quality of life. Our physicians, through their designated practices, have gained worldwide recognition for the successful application of revolutionary minimally invasive stem cell therapies. With over a century of combined medical experience, our doctors have established a patient experience designed with the highest concern for patient safety and quality of care. For more information, visit our website at LungInstitute.com, like us on Facebook, follow us on Twitter or call us today at (855) 469-5864.

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Dr. Keefe on the Role of Supportive Care in Personalized Medicine
Dorothy M. K. Keefe, MD, FRACP, past president, MASCC, director, SA Cancer Service, professor of cancer medicine, University of Adelaide, Australia, discusse...

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Law Offices of Robert E. Gluck, P.A. Plantation, FL., Naples, Florida
http://www.robertgluck.com (877) GluckLaw Robert E Gluck Law Offices. Since becoming a lawyer in 1989 I have handled cases involving the following types of accidents or injuries: Vehicle...

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Recovering from Spinal Cord Injury: Rehabilitation Efforts
Key developments in rehabilitation medicine for those patients who have sustained Spinal Cord Injury (SCI) are presented in a compelling review of end result...

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For many years scientists have been trying to unravel mechanisms that guide function and differentiation of blood stem cells, those cells that generate all blood cells including our immune system. The study of human blood stem cells is difficult because they can only be found in the bone marrow in specialized "niches" that cannot be recapitulated in a culture dish. Now a group of scientists from Dresden led by stem cell researcher Prof. Claudia Waskow (Technische Universitt Dresden) was able to generate a mouse model that supports the transplantation of human blood stem cells despite the species barrier and without the need for irradiation. They used a mutation of the Kit receptor in the mouse stem cells to facilitate the engraftment of human cells.

In the new model human blood stem cells can expand and differentiate into all cell types of the blood without any additional treatment. Even cells of the innate immune system that can normally not be found in "humanized" mice were efficiently generated in this mouse. Of significance is the fact that the stem cells can be maintained in the mouse over a longer period of time compared to previously existing mouse models. These results were now published in the journal Cell Stem Cell.

"Our goal was to develop an optimal model for the transplantation and study of human blood stem cells," says Claudia Waskow, who recently took office of the professorship for "animal models in hematopoiesis" at the medical faculty of the TU Dresden. Before, Prof. Waskow was a group leader at the DFG-Center for Regenerative Therapies Dresden where most of the study was conducted.

The trick used by Claudia Waskow's team to achieve optimal stem cell engraftment was the introduction of a naturally occurring mutation of the Kit receptor into mice that lack a functional immune system. This way they circumvented the two major obstacles of blood stem cell transplantation: the rejection by the recipient's immune system and absence of free niche space for the incoming donor stem cells in the recipient's bone marrow. Space is usually provided by irradiation therapy, called conditioning, because it damages and depletes the endogenous stem cells and thus frees space for the incoming human cells. However, irradiation is toxic to many cell types and can lead to strong side effects. The Kit mutation in the new mouse model impairs the recipient's stem cell compartment in such a way that the endogenous blood stem cells can be easily replaced by human donor stem cells with a functional Kit receptor. This replacement works so efficiently that irradiation can be completely omitted allowing the study of human blood development in a physiological setting. The model can now be used to study diseases of the human blood and immune system or to test new treatment options.

The results from Prof. Waskow's group also show that the Kit receptor is important for the function of human blood stem cells, notably in a transplantation setting. Further studies will now focus on using this knowledge about the role of the receptor to improve conditioning therapy in the setting of therapeutic hematopoietic stem cell transplantation in patients.

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The above story is based on materials provided by Technische Universitt Dresden. Note: Materials may be edited for content and length.

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PUBLIC RELEASE DATE:

10-Jul-2014

Contact: Meng Zhao eic@nrren.org 86-138-049-98773 Neural Regeneration Research

There is evidence that selective serotonin reuptake inhibitor antidepressants can promote neuronal cell proliferation and enhance neuroplasticity both in vitro and in vivo. Dr. Javad Verdi and his team, Tehran University of Medical Sciences, Iran proposed that citalopram, a selective serotonin reuptake inhibitor, can increase the efficacy of bone marrow mesenchymal stem cells (BMSCs) differentiating into neuronal-like cells. Experimental results confirmed that citalopram can improve the neuronal-like cell differentiation of BMSCs by increasing cell proliferation and survival while maintaining their neuronal characteristics. These results were published in Neural Regeneration Research (Vol. 9, No. 8, 2014).

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Article: "Citalopram increases the differentiation efficacy of bone marrow mesenchymal stem cells into neuronal-like cells" by Javad Verdi1, 2, Seyed Abdolreza Mortazavi-Tabatabaei1, 2, Shiva Sharif 2, 3, Hadi Verdi2, Alireza Shoae-Hassani1, 2 (1 Department of Applied Cell Sciences, School of Advanced Technologies in Medicine, Tehran University of Medical Sciences, Tehran, Iran; 2 Department of Stem Cells and Tissue Engineering, Research Center for Science and Technology in Medicine, Tehran University of Medical Sciences, Tehran, Iran; 3 Department of Tissue Engineering, School of Advanced Technologies in Medicine, Iran University of Medical Sciences, Tehran, Iran)

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Meng Zhao eic@nrren.org 86-138-049-98773 Neural Regeneration Research http://www.nrronline.org/

AAAS and EurekAlert! are not responsible for the accuracy of news releases posted to EurekAlert! by contributing institutions or for the use of any information through the EurekAlert! system.

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JUDY WOODRUFF: Theres been a big disappointment in the hope to find a cure for AIDS. It involves a young child who was thought to have been cured of HIV as a baby.

JEFFREY BROWN: It was in March of last year that doctors thought they might have made a breakthrough in the goal of finding a cure for AIDS, treating a baby girl in Mississippi with early and unusually aggressive drug therapy.

The mother had HIV and had not been treated during pregnancy. But the girl was treated within 30 hours of her birth and was free of the virus for two years. Doctors allowed her to stay off therapy and, still, there were no signs of HIV returning.

But, yesterday, officials announced that the girl, now almost 4, had tested positive for HIV during a follow-up visit last week.

Dr. Anthony Fauci of the National Institute of Allergy and Infectious Diseases joins me now.

And welcome back to you.

This is something that you and I talked about when the news came out last year. So, remind us first why this seemed so hopeful, how this early and aggressive treatment promised such a difference.

DR. ANTHONY FAUCI, National Institute of Allergy and Infectious Diseases: Well, it promised such a difference because what happened with this particular baby was an unusual situation, that the baby had been on therapy, this aggressive form that you correctly described, for about 18 months, but then was lost to follow-up.

And the mother discontinued the therapy because she just dropped out of the out of the health care system, came back five months later, and when the physicians examined the baby, they found out that they couldnt find the virus anywhere by the standard methods of looking for virus, no virus in the plasma and no virus in the cells in the blood.

So they decided that this possibly could have been a cure related to the fact that the baby was treated very early, as you mentioned, within 30 hours, and aggressively. As it turned out, they followed the baby very, very carefully, and over a period of 27 months without any therapy at all, there was no indication at all of any virus in the baby. There was no plasma viremia, as we say, namely virus in the blood.

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HIV rebound in young child is another step in long process of AIDS research

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If youre a chimp, the smarts you have are in large part the smarts you were born with, according to a new study that put 99 chimpanzees through a battery of intelligence tests.

The findings, published in the journal Current Biology, show that many cognitive abilities are strongly influenced by genetics and may help shed light on the rise of intelligence in humans.

Research into human IQ has shown that children do indeed inherit their intelligence levels from their parents, the study authors wrote but that intelligence can be modified by nongenetic mechanisms like socioeconomic status. Studying animals, however, can provide insight into the genetic basis of intelligence without those confounding factors that crop up in human cases.

For this study, researchers from Georgia State University in Atlanta took 99 captive chimpanzees ages 9 to 54 and subjected them to 13 different IQ tests spanning a range of different cognitive abilities, from communication skills to spatial reasoning and tool use.

For example, in a sort of shell game, they challenged chimps to remember which of three containers hid a tasty morsel of food. This task tested their spatial memory. Researchers also put some food out of reach and waited to see if the chimps could successfully get a human to help them get it a test of communication skills.

The scientists ran the chimps performance against a number of potential confounding factors, including the animals' sex, age and rearing history and found that genetic differences appeared to account for 54.2% of intelligence a slight majority of a given chimps smarts.

But nature didnt trump nurture in all aspects of intelligence: The genes didnt seem to play a strong role across all of the 13 different cognitive abilities tested. Cognitive abilities involving spatial memory, communication and attention state (among others) seemed significantly inheritable, while skills such as tool use were not.

There could be very good evolutionary reasons that spatial memory and communication skills were inheritable, the study authors wrote.

Presumably, these attributes would have conferred advantages to some individuals, perhaps in terms of enhanced foraging skills or increased social skills, leading to increased opportunities for access to food or mating, the study authors wrote. These individuals would have then potentially had increased survival and fitness, traits that would have become increasingly selected upon during primate evolution.

Follow @aminawrite for more fascinating research from the animal world.

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In-gene-ious? Chimps inherit much of their intelligence, study finds

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PUBLIC RELEASE DATE:

11-Jul-2014

Contact: Jia Liu liujia@genomics.cn BGI Shenzhen

Shenzhen, July 10, 2014---A team of researchers from The Chinese University of Hong Kong, BGI and other institutes have identified a gene of wild soybean linked to salt tolerance, with implication for improving this important crop to grow in saline soil. This study published online in Nature Communications provides an effective strategy to unveil novel genomic information for crop improvement.

Soybean is an important crop for the world. Due to domestication and human selection, cultivated soybeans have less genetic diversities than their wild counterparts. Among the lost genes, some may play important roles for the adaptation to different environments. In this study, scientists used wild soybean as a resource for investigating the valuable genes that adapt to certain environmental conditions.

They sequenced and assembled a draft genome of wild soybean W05, and developed a recombinant inbred population for genotyping-by-sequencing and phenotypic analyses to identify multiple QTLs relevant to traits of interest in agriculture. Using the de novo sequencing data from this work and their previous germplasm re-sequencing data, the team discovered a novel ion transporter gene, GmCHX1, and suggested it maybe related with salt tolerance.

During the following rapid gain-of-function tests, the gene GmCHX1 was conferred its function on salt tolerance, and suggested GmCHX1 acted probably through lowering the Na+/K+ ratio. The authors assumed that the elimination of GmCHX1 in salt-sensitive germplasms may be an example of negative selection against a stress tolerance gene in unstressed environments. The expression of stress tolerance genes may be an energy burden on the plant if the functions of these genes are not required.

Through this study, researchers developed an efficient strategy using the combination of whole-genome de novo sequencing, high-density-marker QTL mapping by re-sequencing, and functional analyses, which could greatly enhance the efficiency of uncovering QTLs and genes for beneficial traits in crop breeding.

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CSULB COE Lecture - Reproducing Body Functions
Over the last several years, engineering has empowered numerous innovations in the field of medicine that have improved the lives of many. These innovations ...

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New to Idaho Waters: Genetics
A look at the concerns of cross breeding and the information learned from the hi-tech tools of genetic research. From our 2007 series, "Idaho Originals,"

By: Idaho Fish Game 75 Years

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The Sims 4: Genetics
I was lucky enough to get early access to The Sims 4 CAS! Here #39;s a little preview of what "play with genetics" looks like. -Regular t.A.T.u. content will resume Saturday! 🙂

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The Sims 4 Create A Sim Traits, Attributes, Genetics
The key to enjoying The Sims games has always come down to how well the creation tools enable you to produce fun and interesting characters to play with in the main game. As with previous...

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Stamford, CT (PRWEB) July 11, 2014

Alliance for Cancer Gene Therapy, Inc. (ACGT) the nations only non-profit dedicated exclusively to cell and gene therapies for cancer has announced that Joshua Corday has joined the organization as Senior Development Officer.

Corday is a veteran fundraising professional with deep experience securing gifts and addressing influential donors. Prior to joining ACGT, he served as Annual Giving Director for NewYork Presbyterian Hospital/Columbia University Medical Center, where he increased Annual Fund revenue and developed a unique fundraising pipeline. Corday has also served as a fundraising professional at Rockland Family Shelter, United Way of Essex and West Hudson, and Special Olympics NY. He holds a BS in Political Science from University of Utah.

Im delighted to join a foundation in the vanguard of treating and combating cancer, Corday said. ACGT is funding alternatives to traditional cancer treatments, and Im proud to be a part of that quest.

ACGT funds top physicians and researchers at medical institutions in the US and Canada with a clear mission top-of-mind: uncovering effective, innovative cancer treatments that supersede radiation, chemotherapy and surgery. ACGT has served as a major funding engine in the fight against cancer since its formation in 2001, and has provided nearly $25 million in grants to date. ACGT was created by Barbara and Edward Netter after the loss of their daughter-in-law to breast cancer. Since Edwards passing in 2011, Barbara Netter has led the foundation as President and Co-Founder, continuing the mission of the organization to focus on groundbreaking treatments for cancer.

With a dearth of public funding, the importance of private philanthropy in supporting cancer research and progress grows daily, said Barbara Netter. Josh is dedicated to expanding this funding avenue he will identify and connect with donors eager to fight cancer and support the worlds best scientists and researchers. ACGT has benefited from the support and belief of private philanthropists since its beginning in 2001, and we look forward to continuing these crucial relationships.

About Alliance for Cancer Gene Therapy (ACGT) Established in 2001, ACGT (http://www.acgtfoundation.org) is the nations only not-for-profit exclusively dedicated to cancer cell and gene therapy treatments for all types of cancer. One-hundred percent of contributions go directly to research. ACGT has funded 44 grants in the U.S. and Canada since its founding in 2001 by Barbara Netter and her late husband, Edward, to conduct and accelerate critically needed innovative research for all types of cancer. ACGTs Scientific Advisory Council is comprised of 16 of the nations most distinguished physicians and researchers in cell and gene therapy, who thoroughly review all grants. Since its inception, ACGT has awarded 29 grants to Young Investigators and 15 grants to Clinical Investigators, totaling $24.7 million in funding. Barbara Netter, as President, together with other members of the Board of Directors, are fully committed to ACGTs continued support of this research. ACGT is located at 96 Cummings Point Road, Stamford, CT 06902.

ACGT on Facebook: http://www.facebook.com/ACGTfoundation ACGT on Twitter: http://www.twitter.com/ACGTfoundation ACGT on YouTube: http://www.youtube.com/user/ACGTfoundation

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Advances in plan for individual gene therapies for leading cause of blindness Scientists use stem cells to investigate causes of degenerative eye disease Two patients with retinitis pigmentosa had mutations in a certain gene New gene therapy was used to rescue vision of mice with the same condition

By Julian Robinson

Published: 06:49 EST, 11 July 2014 | Updated: 08:08 EST, 11 July 2014

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Scientists have moved closer to a breakthrough in 'personalised' treatments for a leading cause of blindness.

Researchers have stepped up their bid to create individual gene therapies for one of the factors that triggers inherited vision loss.

They used 'induced' stem cells - taken from ordinary skin cells - to investigate patient-specific causes of the degenerative eye disease retinitis pigmentosa (RP), which leads to blindness or severe visual impairment.

Scientists have moved closer to a breakthrough in 'personalised' treatments for a leading cause of blindness

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Update

By K.C. Baker

07/10/2014 at 11:30 AM EDT

Cindy Crawford and Chase Foley

Splash News Online; Courtesy Chase Foley

When Cindy Crawford found out about a 2-year-old boy with leukemia who needed a bone marrow transplant to save his life, she had to do something to help.

"I lost my little brother, Jeff, to leukemia when I was just 10 years old," Crawford, 48, tells PEOPLE. "Sadly, a bone marrow transplant wasn't an option for him then."

In 1974, when Crawford was 8 years old, Jeff was diagnosed with acute lymphoblastic leukemia. He was just 2 years old. At the time, she says, the deadly disease only had a 25 percent cure rate. After two years of experimental treatments, Jeff lost his battle, right before his 4th birthday.

The death of her brother, whom she describes as "one of the most influential people in my life," was devastating, says Crawford, who for years has been an advocate for bone marrow donation.

"But today," Crawford explains, "we have the chance to save the life of little Chase Foley and countless other children. We need your help to find him a bone marrow donor."

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Histogen's chief executive, Gail Naughton.

Histogen, a San Diego biotech company developing a hair loss treatment from stem cells, has established a joint venture for cancer therapy.

Privately held Histogen has created the venture, Histogen Oncology, in partnership with the medical device company Wylde LLC. Wylde contributed $2.5 million, said Gail Naughton, the company's chief executive.

The company's technology grows young skin cells called fibroblasts under simulated embryonic conditions, including low oxygen levels. The company says these conditions cause the cells to become embryonic-like, making proteins and substances called growth factors characteristic of young tissue. Histogen uses these substances in its various products.

Histogen Oncology uses certain of these substances that enable cancer cells to undergo programmed cell death, or apoptosis. These substances turn on a gene that controls apoptosis, which naturally occurs in damaged cells, Naughton said.

Since the cancer cells are genetically abnormal, they begin to self-destruct when apoptosis is triggered. Normal cells are not affected, because the apoptosis mechanism is already turned on, she said. The loss of this mechanism is a hallmark of cancer.

Histogen Oncology intends intends to apply within 18 months to start clinical trials in Stage 4 advanced metastatic pancreatic cancer, Naughton said. This cancer is a good target because it has a high mortality rate, so better therapies are urgently needed, she said.

There's an average 6.7 percent survival rate for patients over a five-year period after diagnosis with pancreatic cancer, according to the National Cancer Institute.

"We're hoping that we're going to see an increase in the person's life, without any toxic side effects," Naughton said.

The substances will be given either intravenously or injected into the abdominal cavity.

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Histogen forms cancer joint venture

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PUBLIC RELEASE DATE:

10-Jul-2014

Contact: Lucky Tran lt2549@cumc.columbia.edu 212-305-3689 Columbia University Medical Center

NEW YORK, NY (July 10, 2014) Columbia University Medical Center (CUMC) researchers have created a way to develop personalized gene therapies for patients with retinitis pigmentosa (RP), a leading cause of vision loss. The approach, the first of its kind, takes advantage of induced pluripotent stem (iPS) cell technology to transform skin cells into retinal cells, which are then used as a patient-specific model for disease study and preclinical testing.

Using this approach, researchers led by Stephen H. Tsang, MD, PhD, showed that a form of RP caused by mutations to the gene MFRP (membrane frizzled-related protein) disrupts the protein that gives retinal cells their structural integrity. They also showed that the effects of these mutations can be reversed with gene therapy. The approach could potentially be used to create personalized therapies for other forms of RP, as well as other genetic diseases. The paper was published recently in the online edition of Molecular Therapy, the official journal of the American Society for Gene & Cell Therapy.

"The use of patient-specific cell lines for testing the efficacy of gene therapy to precisely correct a patient's genetic deficiency provides yet another tool for advancing the field of personalized medicine," said Dr. Tsang, the Laszlo Z. Bito Associate Professor of Ophthalmology and associate professor of pathology and cell biology.

While RP can begin during infancy, the first symptoms typically emerge in early adulthood, starting with night blindness. As the disease progresses, affected individuals lose peripheral vision. In later stages, RP destroys photoreceptors in the macula, which is responsible for fine central vision. RP is estimated to affect at least 75,000 people in the United States and 1.5 million worldwide.

More than 60 different genes have been linked to RP, making it difficult to develop models to study the disease. Animal models, though useful, have significant limitations because of interspecies differences. Researchers also use human retinal cells from eye banks to study RP. As these cells reflect the end stage of the disease process, however, they reveal little about how the disease develops. There are no human tissue culture models of RP, as it would dangerous to harvest retinal cells from patients. Finally, human embryonic stem cells could be useful in RP research, but they are fraught with ethical, legal, and technical issues.

The use of iPS technology offers a way around these limitations and concerns. Researchers can induce the patient's own skin cells to revert to a more basic, embryonic stem celllike state. Such cells are "pluripotent," meaning that they can be transformed into specialized cells of various types.

In the current study, the CUMC team used iPS technology to transform skin cells taken from two RP patientseach with a different MFRP mutationinto retinal cells, creating patient-specific models for studying the disease and testing potential therapies.

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Patient-specific stem cells and personalized gene therapy

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Newswise LA JOLLA-The ability to switch out one gene for another in a line of living stem cells has only crossed from science fiction to reality within this decade. As with any new technology, it brings with it both promise--the hope of fixing disease-causing genes in humans, for example--as well as questions and safety concerns. Now, Salk scientists have put one of those concerns to rest: using gene-editing techniques on stem cells doesn't increase the overall occurrence of mutations in the cells. The new results were published July 3 in the journal Cell Stem Cell.

"The ability to precisely modify the DNA of stem cells has greatly accelerated research on human diseases and cell therapy," says senior author Juan Carlos Izpisua Belmonte, professor in Salk's Gene Expression Laboratory. "To successfully translate this technology into the clinic, we first need to scrutinize the safety of these modified stem cells, such as their genome stability and mutational load."

When scientists want to change the sequence of a stretch of DNA inside cells--either for research purposes or to fix a genetic mutation for therapeutic purposes--they have their choice of two methods. They can use an engineered virus to deliver the new gene to a cell; the cell then integrates the new DNA sequence in place of the old one. Or scientists can use what's known as custom targeted nucleases, such as TALEN proteins, which cut DNA at any desired location. Researchers can use the proteins to cut a gene they want to replace, then add a new gene to the mix. The cell's natural repair mechanisms will paste the new gene in place.

Previously, Belmonte's lab had pioneered the use of modified viruses, called helper-dependent adenoviral vectors (HDAdVs) to correct the gene mutation that causes sickle cell disease, one of the most severe blood diseases in the world. He and his collaborators used HDAdVs to replace the mutated gene in a line of stem cells with a mutant-free version, creating stem cells that could theoretically be infused into patients' bone marrow so that their bodies create healthy blood cells.

Before such technologies are applied to humans, though, researchers like Belmonte wanted to know whether there were risks of editing the genes in stem cells. Even though both common gene-editing techniques have been shown to be accurate at altering the right stretch of DNA, scientists worried that the process could make the cells more unstable and prone to mutations in unrelated genes--such as those that could cause cancer.

"As cells are being reprogrammed into stem cells, they tend to accumulate many mutations," says Mo Li, a postdoctoral fellow in Belmonte's lab and an author of the new paper. "So people naturally worry that any process you perform with these cells in vitro--including gene editing--might generate even more mutations."

To find out whether this was the case, Belmonte's group, in collaboration with BGI and the Institute of Biophysics, Chinese Academy of Sciences in China, turned to a line of stem cells containing the mutated gene that causes sickle cell disease. They edited the genes of some cells using one of two HDAdV designs, edited others using one of two TALEN proteins, and kept the rest of the cells in culture without editing them. Then, they fully sequenced the entire genome of each cell from the four edits and control experiment.

While all of the cells gained a low level of random gene mutations during the experiments, the cells that had undergone gene-editing--whether through HDAdV- or TALEN-based approaches--had no more mutations than the cells kept in culture.

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No Extra Mutations in Modified Stem Cells, Study Finds

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Scientists in Missouri have revealed they have induced gender in cells They performed the trick by modifying a gene in multicellular algae This gene was responsible for giving the algae one of two mating types But they altered it so that it could switch between 'male' and female' Could explain origin of the sexes in plant and animal organisms

By Jonathan O'Callaghan

Published: 12:11 EST, 10 July 2014 | Updated: 12:11 EST, 10 July 2014

Throughout evolution, living things have repeatedly developed physically distinct sexes, but how does this actually happen?

Thats the question scientists were hoping to answer when they performed a genetic engineering trick on multicellular algae.

And they were surprised to find the process through which one gender produces eggs and the other sperm was more simple than expected - and the scientists could switch the gender roles of the algae.

Scientists in Missouri have revealed they could induce gender (stock image shown) in cells. They performed the trick by identifying and modifying a gene in multicellular algae. This gene was responsible for giving the algae one of two mating types, but they also made it switch gender

The study, led by Dr James Umen at the Danforth Plant Science Center in Missouri and published in Plos Biology, looked at the multicellular green algae Volvox carteri.

A neuroscientist has claimed the expression 'Men are from Mars and women are from Venus' has no scientific grounding, and that instead our brains are changed by the roles society forces us to play.

According to Gina Rippon, a professor at Aston University in Birmingham, stereotypes - such as women's supposed inability to read maps, or the idea men are bad at multitasking - have no links to science.

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Man, I feel like a woman: Surprisingly simple genetic quirk in transgender algae reveals how separate sexes evolved

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Carlos Castro published in PLOS
M+Visin Fellow Carlos Castro Gonzlez was lead author on a paper published in June 2014 in PLOS Computational Biology. Castro and his team at UPM with other...

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Carlos Castro published in PLOS - Video

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