Gene Therapy News and Research

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Gene therapy is an experimental technique that uses genes to treat or prevent disease. In the future, this technique may allow doctors to treat a disorder by inserting a gene into a patient’s cells instead of using drugs or surgery.

Researchers use patient's own stem cells to correct genetic alteration that causes SCD

Using a patient's own stem cells, researchers at Johns Hopkins have corrected the genetic alteration that causes sickle cell disease (SCD), a painful, disabling inherited blood disorder that affects mostly African-Americans. The corrected stem cells were coaxed into immature red blood cells in a test tube that then turned on a normal version of the gene.

29 Sep 2011

Weill Cornell receives NIH's T-R01 Award for spina bifida research

The National Institutes of Health (NIH) has awarded a five-year, $5.5 million Transformative Research Project (T-R01) Award to fund research into risk factors for spina bifida and related congenital defects in which an area of the affected baby's spine or brain is not fully enclosed.

27 Sep 2011

AGTC receives FFB grant to evaluate gene therapy treatment for X-linked Retinoschisis

Applied Genetic Technologies Corporation, a privately-held, clinical stage biotechnology company developing novel systems to deliver human therapeutics, announces that it has received a grant of $1.5 million from the Foundation Fighting Blindness to fund a pre-clinical trial evaluating the safety and efficacy of a gene therapy treatment for X-linked Retinoschisis (XLRS), a genetic eye disease affecting over 35,000 patients in the US and Europe.

22 Sep 2011

ACT receives UK MHRA clearance for hESC-derived RPE cell trial for Stargardt's Macular Dystrophy

Advanced Cell Technology, Inc., a leader in the field of regenerative medicine, announced today that it has received clearance from the U.K. Medicines and Healthcare products Regulatory Agency to begin treating patients as part of a Phase 1/2 clinical trial for Stargardt's Macular Dystrophy using retinal pigment epithelium (RPE) derived from human embryonic stem cells.

22 Sep 2011

Early ART can be cost-effective and decrease mortality in HIV patients

Researchers at Weill Cornell Medical College and GHESKIO have shown that early treatment of HIV not only saves lives but is also cost-effective. Results are published in today's edition of PLoS Medicine.

22 Sep 2011

Digna Biotech receives FDA Orphan Drug status for cardiotrophin-1 to treat ALF

Digna Biotech, a Spanish biotechnological company, announces that the Food and Drug Administration (FDA) granted cardiotrophin-1 (CT-1) Orphan Drug status for use in the treatment of acute liver failure.

22 Sep 2011

Scientists discover new type of spinal cord stem cell

A group led by a University of British Columbia and Vancouver Coastal Health scientist has discovered a type of spinal cord cell that could function as a stem cell, with the ability to regenerate portions of the central nervous system in people with spinal cord injuries, multiple sclerosis or amyotrophic lateral sclerosis.

16 Sep 2011

Heart specialists reveal how pacemaker works at the biological level

Heart specialists at Johns Hopkins have figured out how a widely used pacemaker for heart failure, which makes both sides of the heart beat together to pump effectively, works at the biological level.

15 Sep 2011

Mary Ann Liebert launches 'Human Gene Therapy Methods' journal

Mary Ann Liebert, Inc. announces the launch of a journal expansion, Human Gene Therapy Methods, to complement the flagship publication Human Gene Therapy.

14 Sep 2011

Cardiotrophin 1 has marked effect on fat and glucose metabolism

Scientists from the Center for Applied Medical Research (CIMA) of the University of Navarra (Spain) have discovered that cardiotrophin 1, a protein synthesized by muscle cells and adipose tissue, has a marked effect on fat and glucose metabolism.

13 Sep 2011

Mayo Clinic develops genome-based immunization strategy against feline AIDS

Mayo Clinic researchers have developed a genome-based immunization strategy to fight feline AIDS and illuminate ways to combat human HIV/AIDS and other diseases. The goal is to create cats with intrinsic immunity to the feline AIDS virus. The findings -- called fascinating and landmark by one reviewer -- appear in the current online issue of Nature Methods.

12 Sep 2011

New treatment strategy for dominant forms of muscular dystrophy

Investigators at The Research Institute at Nationwide Children's Hospital are studying a potential new treatment strategy for dominant forms of muscular dystrophy, thanks to preliminary funding from The Ohio State University Center for Clinical and Translational Science.

12 Sep 2011

Aggressive medical management better than stenting for prevention of recurrent stroke

Patients at a high risk for a second stroke who received intensive medical treatment had fewer strokes and deaths than patients who received a brain stent in addition to the medical treatment, a large nationwide clinical trial has shown. The investigators published the results in today's online first edition of the New England Journal of Medicine.

8 Sep 2011

'Chaperone' protein may play a critical role in pathogenesis of heart failure

Researchers at Mount Sinai School of Medicine have identified a new drug target that may treat and/or prevent heart failure. The team evaluated failing human and pig hearts and discovered that SUMO1, a so-called "chaperone" protein that regulates the activity of key transporter genes, was decreased in failing hearts.

8 Sep 2011

Researchers identify new drug target for heart failure

8 Sep 2011

Biomaterials may heal chronic diabetic wounds

Biomaterials may prove key to healing chronic wounds in Diabetic patients, delegates will hear today at the 24th European Conference on Biomaterials. Over 950 delegates are at the event in Dublin, which is jointly hosted by NUI Galway's Network of Excellence for Functional Biomaterials and the University of Ulster, Jordanstown.

7 Sep 2011

AMT, NIH enter exclusive license agreement for use of AAV5-based gene therapy vectors

Amsterdam Molecular Therapeutics, a leader in the field of human gene therapy, today announced that it has entered into an exclusive license agreement with the US National Institutes of Health (NIH) for use of adeno-associated virus serotype 5 (AAV5)-based gene therapy vectors for liver and brain indications.

6 Sep 2011

UCF, UF receive $5.5 million NIH grant for hemophilia research

At a time when research funding is hard to come by, a University of Central Florida and University of Florida partnership has landed almost $5.5 million in National Institutes of Health highly competitive grants for hemophilia research.

3 Sep 2011

Two ALS-associated genes work in tandem to support long-term survival of motor neurons

Although several genes have been linked to amyotrophic lateral sclerosis (ALS), it is still unknown how they cause this progressive neurodegenerative disease. In a new study, Columbia University Medical Center (CUMC) researchers have demonstrated that two ALS-associated genes work in tandem to support the long-term survival of motor neurons.

2 Sep 2011

CIRM approves stem cell research planning grants for five UC Davis teams

The California Institute for Regenerative Medicine (CIRM), the state stem cell agency, today approved research planning grants for five UC Davis Health System teams that are working to develop human clinical trials to treat illnesses such as Huntington's disease, vascular disease, osteoporosis, HIV/AIDS and airway disease in children.

29 Aug 2011