Archive for the ‘Gene Therapy Research’ Category

Montral (PRWEB) January 23, 2014

Today, with the addition of members in Antarctica, Synbiota Inc. connects synthetic biology researchers across all 7 continents, just 3 months after the company's launch. Also announced today is $300,000 in available funding for Synbiota members and their biological solutions.

"Genetic engineering is our best bet to save the world," says Bill Liao, Founder of CoderDojo, a global network of hackerspaces and partner at SOSVentures. "That's why we're offering Synbiota's global network of researchers and biohackers $300,000 in available funding to accelerate the development of sustainable biological solutions via the SynBio axlr8r program." Learn more, and sign up at synbiota.com/axlr8r.

Synbiota is satisfying a global need for advanced virtual lab technology with its free web-based software. Previously limited to large corporations such as Monsanto, genetic engineering is touted by experts as one of humanity's best tools to combat global challenges in climate, access to food, fuel, materials, and the development of effective, low-cost medicine.

"Biotech is advancing much faster than computer technology, and Synbiota is at the heart of this revolution" says Rob Carlson, principal at Biodesic, and synthetic biology advisor to corporations and governments around the world. "Bringing together a global cadre of independent researchers, and pairing them with real funding opportunities is just the sort of thing that ignites revolutions. This is an incredible opportunity for the iGEM, DIYBio, and entrepreneurial communities to fund their work."

About Synbiota:

Synbiota Inc. was founded in April 2013 with the mission to streamline life science R&D and to make it universally accessible. Synbiota was a Fellow of Mozilla Labs WebFWD, winner of Hacking Healths Most Transformative Technology award, recipient of grants from FedDev and the Eastern Ontario Development Program, and creator of the S PRIZE global biotech contest. Synbiota Inc. has offices at the Digital Media Zone at Ryerson University (DMZ) in Toronto, and Maison Notman in Montral.

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Synbiota Expands Into Antarctica, Attracts Investors

PUBLIC RELEASE DATE:

23-Jan-2014

Contact: Vicki Cohn vcohn@liebertpub.com 914-740-2100 Mary Ann Liebert, Inc./Genetic Engineering News

New Rochelle, NY, January 23, 2013-The U.S. Food and Drug Administration (FDA) mandates adequate enrollment of women in post-approval studies (PAS) of medical devices to ensure that any sex differences in device safety and effectiveness are not overlooked. A group of authors from the FDA report the results of a study evaluating the participation of women and analysis of sex differences in PAS in Journal of Women's Health, a peer-reviewed publication from Mary Ann Liebert, Inc., publishers. The article is available free on the Journal of Women's Health website.

Women may respond differently to medical devices due to factors such as genetics, body size, hormones, or other intrinsic sex-specific or extrinsic societal or environmental factors. They may face greater or lesser risk of adverse events or derive more or less benefit. "Based on these findings, FDA implemented new procedures to ensure participation by sex is evaluated in PAS reviews," state the authors in their article "Enrollment and Monitoring of Women in Post-Approval Studies for Medical Devices Mandated by the Food and Drug Administration."

"It is critically important that we have adequate participation of women in clinical trials, and that we analyze sex differences in study outcomes and adverse events," says Editor-in-Chief Susan G. Kornstein, MD, Executive Director of the Virginia Commonwealth University Institute for Women's Health, Richmond, VA, and President of the Academy of Women's Health.

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About the Journal

Journal of Women's Health, published monthly, is a core multidisciplinary journal dedicated to the diseases and conditions that hold greater risk for or are more prevalent among women, as well as diseases that present differently in women. The Journal covers the latest advances and clinical applications of new diagnostic procedures and therapeutic protocols for the prevention and management of women's healthcare issues. Complete tables of content and a sample issue may be viewed on the Journal of Women's Health website. Journal of Women's Health is the Official Journal of the Academy of Women's Health and the Society for Women's Health Research.

About the Academy

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Are enough women included in medical device studies, as required by the FDA?

Waldo Genetics
Traditional Values. Progressive Practices/ We #39;re a family business. So we think like pork producers, not like a corporation. Everybody here is close to our p...

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Cyprus Institute of Neurology and Genetics|Cyprus School of Molecular Medicine
Take a tour of the Cyprus School of Molecular Medicine. You can read more about CSMM and the institute #39;s degree programs here: http://www.educations.com/Cypr...

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Cyprus Institute of Neurology and Genetics|Cyprus School of Molecular Medicine - Video

Bikini Bottom Genetics 1

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Deer Genetics: Is it #39;Right #39; to Shoot Spikes?
Hiding behind a fallen tree covered in camo paint, a camera guy catches deer hunter Nicole McClain playing "peek-a-boo" with with a spike who stomps his feet...

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Deer Genetics: Is it 'Right' to Shoot Spikes? - Video

[05] Gene Therapy - Let #39;s Play XCOM:Enemy Within
Hello People! Welcome to my new series. XCOM:Enemy Unknown is a tactical squad based game that pits a crack team of soldiers against the greatest threat huma...

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[05] Gene Therapy - Let's Play XCOM:Enemy Within - Video

As part of my 2014 predictions, I argued gene therapy would get hot and investor interest would turn toward the monogenic programs at Sangamo Biosciences (SGMO). The partnership announced on Jan. 9 between Biogen Idec (BIIB) and Sangamo confirmed my prediction to a certain extent, albeit sooner in the year than I expected.

Let's discuss what the Biogen partnership means for Sangamo moving forward, and discuss the implications for other gene therapy stocks like Bluebird (BLUE) and Acceleron (XLRN)?

The partnership covers two Sangamo programs: sickle cell disease (SCD) and beta-thalassemia (BT). Sangamo will receive a $20 million upfront payment and is eligible for just about $300 million in future milestones. While it is not explicitly stated, $15 million of those milestones appear to be related to the start of a phase I trial. Sangamo will receive double-digit royalties on sales and retains the right to co-promote in the U.S., assuming drug approval, of course.

Summed up, the new partnership is a nice entry into the gene therapy business for Biogen and continued validation of the Sangamo monogenic disease pipeline.

Sobek has no position in any stocks mentioned in this column.

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The State of Gene Therapy in a Biogen Idec World: Sangamo, Bluebird and Acceleron

New gene therapy success holds promise for degenerative retinal diseases
Gene therapy researchers at UMass Medical School focused on degenerative retinal diseases are calling a promising new study out of University of Oxford the "...

By: UMass Medical School

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New gene therapy success holds promise for degenerative retinal diseases - Video

PUBLIC RELEASE DATE:

22-Jan-2014

Contact: Kris Van der Beken kris.vanderbeken@vib.be 32-473-7834 VIB (the Flanders Institute for Biotechnology)

A European consortium of epilepsy researchers has reported the discovery of a new gene involved in severe childhood epilepsy. Using a novel combination of technologies, including trio exome sequencing of patient/parental DNA and genetic studies in the tiny larvae of zebrafish, the EuroEPINOMICS RES consortium found that mutations in the gene CHD2 are responsible for a subset of epilepsy patients with symptoms similar to Dravet syndrome a severe form of childhood epilepsy that is in many patients resistant to currently available anti-epileptic drugs. The discovery of CHD2's role in epilepsy offers new diagnostic tools for families and clinicians of children with Dravet syndrome and related genetic epilepsies. In addition, the creation of a zebrafish model for CHD2 encephalopathy may facilitate the discovery of new drugs that can treat patients with this form of epilepsy.

Dravet syndrome is a severe genetic epilepsy with onset during infancy, with initial seizures often triggered by fever. For most Dravet patients these seizures cannot be treated adequately with currently available anti-epileptic drugs, and therefore the syndrome is classified as pharmacoresistant. Dravet patients usually develop moderate to severe cognitive delays and some features of autism, and are at increased risk of SUDEP (sudden unexplained death in epilepsy). Approximately 80% of Dravet patients have mutations in the gene SCN1A which encodes the Nav1.1 sodium channel, however for the remaining 20% of patients the underlying genetic cause has yet to be determined.

To identify novel genes involved in Dravet Syndrome and other genetic epilepsies, epilepsy clinicians and human geneticists across Europe recently initiated the EuroEPINOMICS RES (Rare Epilepsy Syndromes) consortium. In 2011, the EuroEPINOMICS RES consortium was awarded 2,37 million in funding from the national funding agencies participating in the European Science Foundation program to systematically search for novel genes for seizure disorders.

As part of these ongoing research activities, the DNA of Dravet patients without SCN1A mutations was analyzed by trio exome sequencing, which searches across the active parts of the genome for de novo mutations that have arisen in these patients (de novo mutations are DNA copying errors that occur in the parents' gametes or in the fertilized egg or embryo, resulting in the afflicted family member being the first person in their family to have this genetic condition). In a group of 9 such patients, this analysis of their DNA (and the DNA of their parents) resulted in the identification of 2 patients with de novo mutations in CHD2, which stands for chromodomain helicase DNA binding protein 2. A third patient with a CHD2 mutation was subsequently identified as well.

To confirm that mutations in CHD2 cause the epilepsy observed in these patients, the same gene was then functionally analyzed in the tiny larvae of zebrafish, which have emerged in the last decade as a powerful animal model for the study of epilepsy. In the case of CHD2, scientists collaborating with the EuroEPINOMICS RES consortium used antisense technology to rapidly generate zebrafish larvae with a partial loss of function of this gene, and were then able to detect epileptic seizures in these animals using electrographic analysis (this method is very similar to electroencephalography, or EEG, which is used to analyze seizures in humans).

The genetic analysis was led by Peter De Jonghe, head of the Neurogenetics Group of the VIB Department of Molecular Genetics at the University of Antwerp (Antwerp, Belgium) and the epilepsy genetics group in Kiel, headed by Ingo Helbig (Dept. of Neuropediatrics, University of Kiel, Germany). Peter De Jonghe: "This research reinforces our belief that trio sequencing enables us to unravel the genetic background of syndromes which occur spontaneously. Previously, investigations into the genetic causes of syndromes such as Dravet Syndrome were not feasible. These types of investigations were only possible by screening large families and seeing how a disorder was passed along. But in disorders such as Dravet Syndrome, this did not work since the children were so seriously ill that they themselves never went on to have their own children. So this new technology also opens up new perspectives in the search for the genetic background of many disorders."

Ingo Helbig adds that "the epileptic encephalopathies pose a major clinical problem as most children have treatment-resistant epilepsy, intellectual disability and many other medical issues. We hope that identifying the underlying genetic cause will help us find better treatment options for the affected patients. In the past, we were not able to identify the reason why children have severe epilepsy. The discovery of CHD2 as the culprit gene in a subset of children with epileptic encephalopathy is a major step for us."

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European epilepsy consortium identifies new gene for severe childhood epilepsy

PUBLIC RELEASE DATE:

22-Jan-2014

Contact: Vicki Cohn vcohn@liebertpub.com 914-740-2156 Mary Ann Liebert, Inc./Genetic Engineering News

New Rochelle, NY, January 22, 2014Metastatic melanoma has a poor prognosis, but treatment with high-dose interleukin-2 (IL2) can extend survival. Now, a combination of IL2 therapy and activation of patients' immune systems using personalized vaccines made from their own tumor cells has been shown to improve survival rates even more than IL2 alone, according to a new article in Cancer Biotherapy and Radiopharmaceuticals, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. The article is available free on the Cancer Biotherapy and Radiopharmaceuticals website.

"High-Dose IL2 in Metastatic Melanoma: Better Survival in Patients Immunized with Antigens from Autologous Tumor Cell Lines" describes a statistically significant improvement in survival for patients who received IL2 plus tumor cell-based immunotherapy. Authors Robert Dillman, Carol DePriest and Stephanie McClure, Hoag Institute for Research and Education, Hoag Family Cancer Institute, and Cancer Biotherapy Research Group, Newport Beach, CA, found that administration of immunotherapy after IL2 treatment resulted in longer patient survival than if individuals were vaccinated before receiving IL2.

"This is an important addition to the literature on IL2 treatment for metastatic melanoma demonstrating that personalized vaccine therapy contributed to an increased survival rate," says Co-Editor-in-Chief Donald J. Buchsbaum, PhD, Division of Radiation Biology, Department of Radiation Oncology, University of Alabama at Birmingham.

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About the Journal

Cancer Biotherapy and Radiopharmaceuticals, published 10 times a year in print and online, is under the editorial leadership of Editors Donald J. Buchsbaum, PhD and Robert K. Oldham, MD, Lower Keys Cancer Center, Key West, FL. Cancer Biotherapy and Radiopharmaceuticals is the only journal with a specific focus on cancer biotherapy, including monoclonal antibodies, cytokine therapy, cancer gene therapy, cell-based therapies, and other forms of immunotherapy. The Journal includes extensive reporting on advancements in radioimmunotherapy and the use of radiopharmaceuticals and radiolabeled peptides for the development of new cancer treatments. Topics include antibody drug conjugates, fusion toxins and immunotoxins, nanoparticle therapy, vascular therapy, and inhibitors of proliferation signaling pathways. Tables of content and a sample issue are available on the Cancer Biotherapy and Radiopharmaceuticals website.

About the Publisher

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Can personalized tumor vaccines improve interleukin-2 treated metastatic melanoma?

PUBLIC RELEASE DATE:

21-Jan-2014

Contact: Sophie Mohin smohin@liebertpub.com 914-740-2100 x2254 Mary Ann Liebert, Inc./Genetic Engineering News

New Rochelle, January 21, 2014 Too much information can be overwhelming, but when it comes to certain types of data that are used to build predictive models to guide decision making there is no such thing as too much data, according to an article in Big Data, the highly innovative, peer-reviewed journal from Mary Ann Liebert, Inc., publishers. The article is available on the Big Data website.

To determine whether more data is really better for predictive modeling, Enric Junqu de Fortuny and David Martens, University of Antwerp, Belgium, and Foster Provost, New York University, NY, tested nine different applications in which they built models using a particular type of data called fine-grained data, such as observing an individual's behavior in a certain setting. In the article "Predictive Modeling with Big Data: Is Bigger Really Better?" the authors state that "certain telling behaviors may not be observed in sufficient numbers without massive data."

"The power of any analytic tool is in using it appropriately," says Founding Editor, Edd Dumbill. "Sweeping assumptions such as 'bigger is better' can be dangerous. This paper significantly advances our knowledge of when massive datasets improve decision-making ability."

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About the Journal

Big Data, published quarterly in print and online, facilitates and supports the efforts of researchers, analysts, statisticians, business leaders, and policymakers to improve operations, profitability, and communications within their organizations. Spanning a broad array of disciplines focusing on novel big data technologies, policies, and innovations, the Journal brings together the community to address the challenges and discover new breakthroughs and trends living within this information.

About the Publisher

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Bigger (data) is better and can improve decision making

AP/January 22, 2014

CONCORD, N.H. (AP) New Hampshires House killed a bill Wednesday that would have required genetically modified foods to be labeled.

The House voted 185-162 to kill the bill, despite supporters arguments it is time for states like New Hampshire to lead on the issue regardless of the federal governments position.

Supporters argued New Hampshire residents have a right to know whether their food is produced with genetic engineering, but critics said the federal Food and Drug Administration has not mandated the labeling because it determined the foods are safe.

The reality is most of us are living every day with the benefits of genetic engineering, said Rep. Linda Lauer, D-Bath. She said insulin has been genetically engineered since 1982. Prior to that insulin was taken from the pancreas of farm animals, she said.

Lauer said the labeling required under the bill would not tell consumers what was in the food, only that it had been genetically engineered. She said the label wouldnt provide accurate information about the foods. For example, genetically engineered beets are used to produce sugar, which is a pure chemical compound. Despite its purity, any foods containing the sugar would have to be labeled, she said.

But Rep. Peter Bixby, D-Dover, said people have a right to know if genetic engineering modified the foods.

People are responsible for their own decisions, but to make those decisions they need information, he said.

But opponents said wary consumers could buy organic foods or foods labeled as not being genetically modified. They said the industry is beginning to respond to consumers wishes for genetically engineered foods to be labeled,

The market will solve this problem. It moves a little slow, but it will solve the problem, said Rep. Robert Haefner, R-Hudson.

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House rejects genetically modified foods labeling

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Newswise A new study conservatively estimates that one in five women with ovarian cancer has inherited genetic mutations that increase the risk of the disease, according to research at Washington University School of Medicine in St. Louis.

Most women in the study would have been unaware of a genetic predisposition to ovarian cancer because they didnt have strong family histories that suggested it.

The research, published Jan. 22 in Nature Communications, is the first large-scale analysis of the combined contributions of inherited and acquired mutations in a major cancer type. The inherited mutations by themselves are unlikely to cause ovarian cancer but may conspire with other genetic changes acquired over a womans lifetime to tip the balance toward cancer, the researchers said.

Earlier studies that have looked at inherited susceptibility to ovarian cancer have focused on women with known family histories of the disease. For the current study, however, the researchers studied 429 women with ovarian cancer that appeared to develop sporadically, meaning the women did not have known family histories of the disease.

Using advanced genomic analysis, we found that 20 percent of women with ovarian cancer had inherited mutations in a gene pathway known to be important in inherited breast and ovarian cancer. That number seems pretty high, explained senior author Li Ding, PhD, assistant director at The Genome Institute at the School of Medicine and a research member of Siteman Cancer Center. This tells us that we need to find better ways to screen women for ovarian cancer, even if they dont have family histories of the disease.

Ovarian cancer strikes an estimated 22,000 women annually. Its symptoms are nonspecific and include bloating, pelvic pain and frequently feeling the need to urinate. Most women arent diagnosed until the cancer has spread, leading to a poor five-year survival rate of 43 percent.

Women with ovarian cancer in the study did not have known family histories of breast or ovarian cancer or rare cancer syndromes, all of which can increase the odds of developing ovarian tumors. The women ranged in age from 26 to 89, and 90 percent were Caucasian.

The researchers, including Washington University first authors Krishna Kanchi, Kimberly Johnson, PhD, and Charles Lu, PhD, performed a genetic analysis of each womans tumor and her own DNA, taken from a skin sample. By comparing the genetic sequences side-by-side, they identified the acquired mutations in individual tumor samples. In addition, by comparing the patients DNA samples with the DNA of 557 women who did not have ovarian cancer and served as controls, the researchers found inherited mutations.

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Study Shows 1 in 5 Women with Ovarian Cancer Has Inherited Predisposition

Population genetics, Cystic Fibrosis - problem solving
Cystic fibrosis is a genetic disorder that affects the lungs and causes nutritional deficiencies. It is caused by a defective recessive gene. This means you ...

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-21- Let #39;s Play Xcom Enemy Within - Genetics Vs MEC #39;s
Xcom Enemy Within Playlist - https://www.youtube.com/watch?v=1jge6hongP4 list=PLJzQm-qJ_UmCdL_KTCT25syHKw_3jTw9n Feel free to like comment and subscribe for ...

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RussoBioClass Basic Genetics

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RussoBioClass Basic Genetics - Video

PUBLIC RELEASE DATE:

22-Jan-2014

Contact: Kathy Ridgely Beal kbeal@acmg.net 301-238-4582 American College of Medical Genetics

Do you cover genetics, genomics, healthcare or medicine? The media are invited to register now for the American College of Medical Genetics and Genomics Annual Clinical Genetics Meeting, March 25-29, 2014 at the Nashville Convention Center.

From Incidental Findings to Whole Genome/Exome Sequencing to Cancer Genetics, the focus of the ACMG Meeting is on the actual practice of genetics and genomics in healthcare, showcasing the latest breakthroughs in genetics research and its practical applications to medical practice. The ACMG Annual Meeting attracts medical and scientific leaders from around the world working to apply research in the human genome to the diagnosis, management, treatment and prevention of genetic conditions and rare and common diseases.

Reporters will hear about the latest medical genetics research; have the opportunity to interact with doctors, laboratory professionals and genetic counselors about what is happening right now in genetics and genomics; and view the latest products available in the extensive exhibit hall.

Topics range from common conditions to rare diseases. Sessions include information of interest to the general public, to health professionals and to the industry/trade.

The ACMG Meeting is the genetics meeting most focused on the practical applications of genetic discoveries in the clinical setting. And the 2014 Meeting is already shattering records with a record number of abstracts submitted and attendee registration to date is at an all-time high.

Two Genetics Short Courses on Tuesday, March 25:

Program Highlights:

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Complimentary press registration now open for ACMG 2014 Annual Clinical Genetics Meeting

Clare McLean

Gene therapy researcher Martin K. Childers with his family dog, Bella, who carries the gene for the disorder he studies.

Preclinical studies show that gene therapy can improve muscle strength in small- and large-animal models of a fatal congenital childhood disease know as X-linked myotubular myopathy.

The findings, appearing in the January 22, 2014 issue of Science Translational Medicine, also demonstrate the feasibility of future clinical trials of gene therapy for this devastating disease.

Watch a video by Brian Donohue on this study.

Researchers at the University of Washington, Gnthon in France, Boston Childrens Hospital, and Virginia Polytechnic Institute and State University in Blacksburg, Va., conducted the study.

The study was based on seminal work on local and systemic administration in a mouse model of the disease performed by Anna Buj-Bello, at Gnthon since 2009. The UWs Martin K. Childers, working with Buj-Bello and Beggs groups, tested gene therapy using an engineered adenovirus vector, created by Gnthon. The vector carries a replacement MTM1 gene.

They used two animal models: mice with an engineered MTM1 mutation and dogs carrying a naturally occurring MTM1 gene mutation. These mutant animals appear very weak with shortened lifespans, similar to patients with myotubular myopathy.

The scientists found that both mice and dogs responded to a single intravascular injection of an adenovirus vector engineered for gene replacement therapy, produced at Gnthon. The treated animals had robust improvement in muscle strength, corrected muscle structure at the microscopic level, and prolonged life. No toxic or immune response was observed in the dogs.

These results demonstrate the efficacy of gene replacement therapy for myotubular myopathy in animal models and pave the way to a clinical trial in patients.

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Gene therapy leads to robust improvements in animal model ...

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Explore the what's and why's of gene therapy research, includingan in-depth look at the genetic disorder cystic fibrosis and how gene therapy could potentially be used to treat it.

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Explore the methods for delivering genes into cells.

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You are the doctor! Design and test gene therapy treatments with ailing aliens.

Supported by a Science Education Partnership Award (SEPA) Grant No. R25RR016291 from the National Center for Research Resources, a component of the NIH. The contents provided here are solely the responsibility of the authors and do not necessarily represent the official views of NIH.

APA format: Genetic Science Learning Center (2014, January 14) Gene Therapy. Learn.Genetics. Retrieved January 22, 2014, from http://learn.genetics.utah.edu/content/genetherapy MLA format: Genetic Science Learning Center. "Gene Therapy." Learn.Genetics 22 January 2014 <http://learn.genetics.utah.edu/content/genetherapy> Chicago format: Genetic Science Learning Center, "Gene Therapy," Learn.Genetics, 14 January 2014, <http://learn.genetics.utah.edu/content/genetherapy> (22 January 2014)

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Learn Genetics - Gene Therapy

SCIENTISTS in Florida, USA, are studying gene therapy to cure genetic eye diseases.

Prof. Galileo Encabo, senior biological scientist at the Department of Opthalmology of the University of Florida, said they have conducted successful human clinical trials, but it would take years before the technology can be made available to the public.

From clinical trials, it takes a few years to make it available for public use. We will have to find a pharmaceutical firm willing to produce (the technology), then we have to get approval from the different bureaucrats, Encabo told reporters.

Gene therapy is the introduction of new genes into human cells to treat a disease. It involves replacing, correcting or removing an abnormal gene.

Encabo held a seminar at the University of the Philippines Cebu yesterday. He was a graduate and a former professor of the university.

The two-hour seminar, held at the UP Cebu Union building, was organized by the UP Cebu Central Visayas Studies Center in partnership with the universitys Sciences Cluster.

It aims to inspire students to pursue studies on molecular genetics.

The University of Florida is collaborating with 65 other institutions in its research on gene therapy.

Encabo said the persons who were subjected to the clinical trials improved their vision, with some increasing their visual ability 10,000 times.

The success of the human clinical trials, he said, will attract more private organizations to support the experiments and validate the whole concept of human gene therapy.

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Pinoy scientist studies genes to treat bad eyes

PUBLIC RELEASE DATE:

22-Jan-2014

Contact: Kim Blakely krb13@uw.edu 206-685-1323 University of Washington

Preclinical studies show that gene therapy can improve muscle strength in small- and large-animal models of a fatal congenital pediatric disease known as X-linked myotubular myopathy. The results, appearing in the Jan. 22 issue of Science Translational Medicine, also demonstrate the feasibility of future clinical trials of gene therapy for this devastating disease.

Researchers, based at the University of Washington, Seattle, Washington, Gnthon, France, Boston Children's Hospital in Massachusetts, and Virginia Polytechnic Institute and State University (Virginia Tech) in Blacksburg, Virginia, conducted the study. The scientists found that both mice and dogs responded from a single intravascular injection of AAV, produced at Gnthon, with robust improvement in muscle strength, corrected muscle structure at the microscopic level, and prolonged life. No toxic or immune response was observed in the dogs.

These results demonstrate the efficacy of gene replacement therapy for myotubular myopathy in animal models and pave the way to a clinical trial in patients.

Children born with X-linked myotubular myopathy, which affects about 1 in 50,000 male births, have very weak skeletal muscles, causing them to appear floppy, with severe respiratory difficulties. Survival beyond birth requires intensive support, often including tube feeding and mechanical ventilation, but effective therapy is not available for patients, and most die in childhood.

Alan H. Beggs of Boston Children's Hospital, co-senior author on the paper, has studied the mutated gene, known as MTM1, for many years and previously showed that replacing missing myotubularin protein effectively improved MTM muscles' ability to contract.

Based on seminal work on local and systemic administration in a mouse model of the disease performed by Anna Buj-Bello at Gnthon since 2009, Martin K. Childers, a professor of rehabilitation medicine and a regenerative medicine researcher at the University of Washington, worked with the Buj-Bello and Beggs groups.

They tested gene therapy using an engineered adenovirus vector, created by Gnthon. The vector is a vehicle for delivering a replacement MTM1 gene into cells. The researchers used two animal models: mice with an engineered MTM1 mutation and dogs carrying a naturally occurring MTM1 gene mutation. These mutant animals appear very weak with shortened lifespans, similar to patients with myotubular myopathy.

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Gene therapy leads to robust improvements in animal model of fatal muscle disease

Cell Therapy: 1-year Results CADUCEUS
CSWN talks with Eduardo Marban, MD, PhD, of the Cedars Sinai Heart Institute in Los Angeles, about the 1-year CADUCEUS results. This interview was conducted ...

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Cell Therapy: 1-year Results CADUCEUS - Video

Pluristem Therapeutics Inc. (PSTI), the Israeli developer of stem-cell therapies, rose the most in more than 17 months after an experimental treatment showed promise in a study of 20 patients with muscle injuries.

The stock surged 22 percent to 16.18 shekels ($4.63) at 11:04 a.m. in Tel Aviv. Earlier it gained as much as 27 percent, the biggest increase since Aug. 6, 2012. The shares fell 15 percent yesterday ahead of the study results.

The early-stage clinical trial assessing Pluristems placental-expanded, or PLX-PAD, cells in people who had a buttock muscle injured during hip-replacement surgery found the treatment was safe, the company said in a statement today. Patients getting the injection also fared better in a muscle-contraction exercise six months later.

These are remarkable results that signal advances in the cell-therapy industry, Jason Kolbert, an analyst with Maxim Group LLC in New York, said at a press conference organized by Pluristem in Tel Aviv.

The study results suggest the stem-cell therapy could help treat a broader range of muscle and tendon injuries, according to the Haifa-based company. We intend to move forward with implementing our strategy towards using PLX cells in orthopedic indications and muscle trauma, Chief Executive Officer Zami Aberman said in the statement.

The results come after the U.S. Food and Drug Administration in June placed one of Pluristems most advanced studies on hold after a patient suffered an allergic reaction. The hold was lifted in September.

To contact the reporter on this story: David Wainer in Tel Aviv at dwainer3@bloomberg.net

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Pluristem Gains Most in 17 Months on Stem-Cell Study

Monsantos new veggies are sweeter, crunchier, and more nutritiouswith none of the Frankenfoods ick factor.

In a windowless basement room decorated with photographs of farmers clutching freshly harvested vegetables, three polo-shirt-and-slacks-clad Monsanto executives, all men, wait for a special lunch. A server arrives and sets in front of each a caprese-like saladtomatoes, mozzarella, basil, lettuceand one of the execs, David Stark, rolls his desk chair forward, raises a fork dramatically, and skewers a leaf. He takes a big, showy bite. The other two men, Robb Fraley and Kenny Avery, also tuck in. The room fills with loud, intent, wet chewing sounds.

Eventually, Stark looks up. Nice crisp texture, which people like, and a pretty good taste, he says.

Its probably better than what I get out of Schnucks, Fraley responds. Hes talking about a grocery chain local to St. Louis, where Monsanto is headquartered. Avery seems happy; he just keeps eating.

The men poke, prod, and chew the next course with even more vigor: salmon with a relish of red, yellow, and orange bell pepper and a side of broccoli. The lettuce is my favorite, Stark says afterward. Fraley concludes that the pepper changes the game if you think about fresh produce.

Changing the agricultural game is what Monsanto does. The company whose nameis synonymous with Big Ag has revolutionized the way we grow foodfor better or worse. Activists revile it for such mustache-twirling practices as suing farmers who regrow licensed seeds or filling the world with Roundup-resistant superweeds. Then theres Monsantos reputationscorned by some, celebrated by othersas the foremost purveyor of genetically modified commodity crops like corn and soybeans with DNA edited in from elsewhere, designed to have qualities nature didnt quite think of.

So its not particularly surprising that the company is introducing novel strains of familiar food crops, invented at Monsanto and endowed by their creators with powers and abilities far beyond what you usually see in the produce section. The lettuce is sweeter and crunchier than romaine and has the stay-fresh quality of iceberg. The peppers come in miniature, single-serving sizes to reduce leftovers. The broccoli has three times the usual amount of glucoraphanin, acompound that helps boost antioxidant levels. Starks department, the global trade division, came up with all of them.

Grocery stores are looking in the produce aisle for something that pops, that feels different, Avery says. And consumers are looking for the same thing. If the team is right, theyll know soon enough. Frescada lettuce, BellaFina peppers, and Benefort broccolicheery brand names trademarked to an all-but-anonymous Monsanto subsidiary called Seminisare rolling out at supermarkets across the US.

But heres the twist: The lettuce, peppers, and broccoliplus a melon and an onion, with a watermelon soon to followarent genetically modified at all. Monsanto created all these veggies using good old-fashioned crossbreeding, the same technology that farmers have been using to optimize crops for millennia. That doesnt mean they are low tech, exactly. Starks division is drawing on Monsantos accumulated scientific know-how to create vegetables that have all the advantages of genetically modified organisms without any of the Frankenfoods ick factor.

And thats a serious business advantage. Despite a gaping lack of evidence that genetically modified food crops harm human health, consumers have shown a marked resistance to purchasing GM produce (even as they happily consume products derived from genetically modified commodity crops). Stores like Whole Foods are planning to add GMO disclosures to their labels in a few years. State laws may mandate it even sooner.

Continue reading here:
What Happens When Monsanto, the Master of Genetic Modification, Decides to Take Nature’s Path?