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.

State highlights: 'Habilitation' an essential benefit, but what is it?; Va. gov. candidates differ on mental health system; Calif. pays $2.7M for late hearings

A selection of health policy stories from Virginia, California, Colorado, Missouri, Pennsylvania, Kansas and Texas.

25 Oct 2013

Researchers use nanopores to smuggle DNA or proteins across membranes

A living cell is built with barriers to keep things out - and researchers are constantly trying to find ways to smuggle molecules in.‬ ‪Professor Giovanni Maglia (Biochemistry, Molecular and Structural Biology, KU Leuven) and his team have engineered a biological nanopore that acts as a selective revolving door through a cell's lipid membrane. The nanopore could potentially be used in gene therapy and targeted drug delivery.‬‬‬‬‬‬‬

24 Oct 2013

Research Institute receives Fast Track status for its gene therapy product for treatment of SMA

AveXis and BioLife, synthetic biology platform companies, today announced that The Research Institute at Nationwide Children's Hospital received Fast Track designation from the U.S. Food and Drug Administration for its scAAV9.CB.SMN gene therapy product for the treatment of spinal muscular atrophy (SMA).

19 Oct 2013

Researchers identify C. perfringens type B bacteria believed to trigger multiple sclerosis

A research team from Weill Cornell Medical College and The Rockefeller University has identified a bacterium it believes may trigger multiple sclerosis, a chronic, debilitating disorder that damages myelin forming cells in the brain and spinal cord.

18 Oct 2013

Detroit Lions, Josephine Ford Cancer Institute to raise $15M for cancer research in metro Detroit

The Detroit Lions and Josephine Ford Cancer Institute, Michigan's largest provider of adult cancer care, have created a unique partnership with the goal to raise $15 million during the next three years for cancer research in metro Detroit.

16 Oct 2013

Ghrelin hormone released during chronic stress may predispose people to PTSD

About a dozen years ago, scientists discovered that a hormone called ghrelin enhances appetite. Dubbed the "hunger hormone," ghrelin was quickly targeted by drug companies seeking treatments for obesity - none of which have yet panned out.

16 Oct 2013

New gene transfer strategy effective for achieving persistent immunotherapy to treat tumors

Cancer immunotherapy can successfully use the body's own immune system to kill tumor cells. But some current approaches to stimulate an antitumor immune response are short-lived, with limited clinical effectiveness. A new gene transfer strategy that introduces modified, immune-stimulating human stem cells is both feasible and effective for achieving persistent immunotherapy to treat leukemias and lymophomas, according to a study published in Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers.

11 Oct 2013

Combining gene therapy with stem cells leads to faster healing of burns

Johns Hopkins researchers, working with elderly mice, have determined that combining gene therapy with an extra boost of the same stem cells the body already uses to repair itself leads to faster healing of burns and greater blood flow to the site of the wound.

10 Oct 2013

Oncosec Medical reports encouraging results from combination study on aggressive melanoma

OncoSec Medical Inc., a company developing its advanced-stage ImmunoPulse DNA-based immunotherapy and NeoPulse therapy to treat solid tumors, has announced positive preliminary animal data demonstrating the benefits of combining the company's ImmunoPulse with anti-CTLA4 and anti-PD1 antibodies.

9 Oct 2013

Studies suggest infusion of blood vessel cells may contribute to organ regeneration in future

Damaged or diseased organs may someday be healed with an injection of blood vessel cells, eliminating the need for donated organs and transplants, according to scientists at Weill Cornell Medical College.

9 Oct 2013

Small alteration in a brain gene contributes to risk for anxiety, depression and memory loss

Researchers at Weill Cornell Medical College have discovered why a tiny alteration in a brain gene, found in 20 percent of the population, contributes to the risk for anxiety, depression and memory loss.

20 Sep 2013

Overexpression of cytoglobin gene inhibits hypoxic injury to neuroblastoma cells

Cytoglobin is a temporary oxygen reservoir, which might provide a minimal, but continuous supply of intracellular oxygen during ischemic and anoxic conditions.

17 Sep 2013

Stem cells and gene therapy play important role in regeneration of inner ear hair cells

Since Moffat and Ramsden for the first time discovered the possibility of the auditory system in humans in 1977, over the last two decades, great progress has been made in physiopathological research on neurosensory hearing loss. J-rgensen and Mathiesen were the first authors to note the capacity for regeneration of the normal vestibular epithelium in adult Australian parrots.

13 Sep 2013

Research holds promise for developing treatments for retinitis pigmentos

One of the leading causes of inherited blindness in humans, retinitis pigmentosa, is analogous to retinal dystrophies that lead to blindness in dogs. Because of their similarities, identifying gene mutations underlying several types of canine retinal dystrophies has suggested courses for research on RP in humans. These advances are significant for both humans and dogs.

12 Sep 2013

New HIV/AIDS vaccine candidate appears to clear AIDS-causing virus from body

An HIV/AIDS vaccine candidate developed by researchers at Oregon Health & Science University appears to have the ability to completely clear an AIDS-causing virus from the body.

12 Sep 2013

Weill Cornell Medical College gets $100M gift to launch Driving Discoveries, Changing Lives campaign

Weill Cornell Medical College announced today that it has received a $100 million gift from longtime benefactors Joan and Sanford I. Weill and the Weill Family Foundation to launch the Medical College's $300 million Driving Discoveries, Changing Lives campaign dedicated to using the most advanced scientific approaches to rapidly translate research breakthroughs into innovative treatments and therapies for patients.

10 Sep 2013

One-time treatment slows onset and progression of ALS, study finds

Studies of a therapy designed to treat amyotrophic lateral sclerosis suggest that the treatment dramatically slows onset and progression of the deadly disease, one of the most common neuromuscular disorders in the world.

10 Sep 2013

Research findings raise hope for new heart therapies

Many people who survive a heart attack find themselves back in the hospital with a failing heart just years later. And the outcome often is unfavorable, owing to limited treatment options. But scientists at Temple University School of Medicine's Cardiovascular Research Center recently found hope in an unlikely source - stem cells in cortical, or compact, bone.

5 Sep 2013

UCLA researchers receives $13M Early Translational grants to advance innovative discoveries using stem cells

Four researchers from UCLA's Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research have received Early Translational Research Awards totaling approximately $13 million from the California Institute for Regenerative Medicine, the state's stem cell agency. The UCLA researchers received four of the 12 total awards; no other institution received more than one.

4 Sep 2013

Argininosuccinic aciduria (ASA) and gene therapy: an interview with Dr Julien Baruteau, UCL Institute for Women's Health, London

This rare genetic condition can theoretically affect patients at any age but symptoms are more often observed in childhood and even uncommonly during the first days of life.

2 Sep 2013