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.

Manchester scientists treat fatal genetic brain disease using stem cell gene therapy

Scientists from The University of Manchester have used stem cell gene therapy to treat a fatal genetic brain disease in mice for the first time.

From The University of Manchester 25 Jul 2013

Investigators report complete catalog of genes expressed in retina

Investigators at Massachusetts Eye and Ear and Harvard Medical School have published the most thorough description of gene expression in the human retina reported to date. In a study published today in the journal BMC Genomics, Drs. Michael Farkas, Eric Pierce and colleagues in the Ocular Genomics Institute at Mass. Eye and Ear reported a complete catalog of the genes expressed in the retina.

20 Jul 2013

Researchers identify cellular channels critical for hearing

Ending a 30-year search by scientists, researchers at Boston Children's Hospital have identified two proteins in the inner ear that are critical for hearing, which, when damaged by genetic mutations, cause a form of delayed, progressive hearing loss. Findings were published online July 18 by the journal Neuron.

19 Jul 2013

Audentes Therapeutics closes $30 million Series A financing

Audentes Therapeutics, Inc., a recently founded biotechnology company dedicated to the development of innovative treatments for rare muscle diseases, today announced the closing of a $30 million Series A financing.

18 Jul 2013

CHOP-led research increases numbers of patients who could be treated with gene therapy

Gene therapy researchers have produced a bioengineered decoy that fools the immune system and prevents it from mistakenly defeating the benefits delivered by a corrective gene.

18 Jul 2013

Scientists find way to sneak nanoparticles carrying anticancer drugs past cells of immune system

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17 Jul 2013

Smokers who seem healthy have damaged airway cells similar to cells found in aggressive lung cancer

Smokers who've received a clean bill of health from their doctor may believe cigarettes haven't harmed their lungs. However, researchers at Weill Cornell Medical College have found that even smokers who seem healthy have damaged airway cells, with characteristics similar to cells found in aggressive lung cancer.

17 Jul 2013

Weill Cornell reports new findings from IVIG Phase 3 trial in Alzheimer's disease at AAIC 2013

Weill Cornell Medical College neurologist Dr. Norman Relkin reported new findings today from the Phase 3 clinical trial of IVIG (intravenous immunoglobulin) in mild to moderate Alzheimer's disease at the Alzheimer's Association International Conference in Boston, Mass.

16 Jul 2013

'Defanged' HIV used to deliver gene therapy

Researchers are having success using a "defanged" version of HIV to deliver gene therapy to children suffering from genetic diseases, a development that could improve other such care.

15 Jul 2013

First Edition: July 12, 2013

Today's early morning highlights from the major news organizations, including articles on data tracking of doctors and comparing the rollout of the Medicare Part D drug plans to implementation of the federal health law.

12 Jul 2013

Hydrogel method form synthetic scaffolds to support formation of blood vessels in engineered tissues

Next-generation hydrogels can form synthetic scaffolds to support the formation of replacement tissues and organs in the emerging area of regenerative medicine. Embedding peptides into the hydrogels stimulates the growth of essential microvascular networks to ensure a good blood supply.

12 Jul 2013

Research on activating Glial Cells to be presented at 7th Annual Pain Therapeutics Summit East

Linda Watkins, Ph.D., Distinguished Professor, Department of Psychology & Neuroscience and Center for Neuroscience, University of Colorado, Co-Chair, Advisory Board, Xalud Therapeutics, Inc. will present research on activating Glial Cells as part of the speaking faculty at the 7th Annual Pain Therapeutics Summit East in Boston, MA.

11 Jul 2013

Action Medical Research funds three new Research Training Fellowships

Action Medical Research, the UK-wide charity funding vital research to help sick and disabled babies and children, has today announced it is funding three new Research Training Fellowships worth more than £470,000.

9 Jul 2013

3-gene signature test will improve care of kidney transplant patients

A breakthrough non-invasive test can detect whether transplanted kidneys are in the process of being rejected, as well as identify patients at risk for rejection weeks to months before they show symptoms, according to a study published in The New England Journal of Medicine (NEJM).

4 Jul 2013

Gene therapy developed by UAB cures pediatric Sanfilippo Syndrome A in animal models

A single session of a gene therapy developed by the Universitat Autònoma de Barcelona (UAB) cures Sanfilippo Syndrome A in animal models. This syndrome is a neurodegenerative disease that affects between 1 and 9 out of every 100,000 children, and causes the death of the child on reaching adolescence.

3 Jul 2013

UCLA researchers introduce gene therapy technique to treat sickle cell disease

Researchers at UCLA's Eli & Edythe Broad Center of Regenerative Medicine & Stem Cell Research have successfully established the foundation for using hematopoietic (blood-producing) stem cells from the bone marrow of patients with sickle cell disease to treat the disease.

2 Jul 2013

Fondation Leducq grants $6M to cardiovascular scientists to help advance cardiac regeneration therapeutics

The Fondation Leducq in Paris, France, dedicated to improving human health through international efforts to combat cardiovascular disease, awarded a $6 million grant award to a new global research network of cardiovascular scientists which includes three researchers from the Cardiovascular Research Center at Icahn School of Medicine at Mount Sinai.