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.

Weill Cornell and NewYork-Presbyterian create pioneering Institute for Precision Medicine

Recognizing that medicine is not "one size fits all," Weill Cornell Medical College and NewYork-Presbyterian Hospital have created the pioneering Institute for Precision Medicine at Weill Cornell and NewYork-Presbyterian/Weill Cornell Medical Center. This new, cutting-edge translational medicine research hub will explore the new frontier of precision medicine, offering optimal targeted, individualized treatment based on each patient's genetic profile.

1 Feb 2013

Insight into reasons why precocious puberty occurs in females

New research from Oregon Health & Science University has provided significant insight into the reasons why early-onset puberty occurs in females.

30 Jan 2013

New study may help treat patients with multiple myeloma

A study led by Robert G. Hawley, Ph.D., professor and chair of the department of anatomy and regenerative biology at the George Washington University (GW) School of Medicine and Health Sciences (SMHS), may help predict which patients with multiple myeloma will respond better to certain treatments.

25 Jan 2013

Researchers initiate first gene therapy clinical trial for Fabry disease

Researchers in Calgary have launched the first gene therapy clinical trial in the world for Fabry disease, a rare inherited enzyme deficiency that can shorten the lifespan of people who have it by as much as 40 years.

24 Jan 2013

Study may help predict which multiple myeloma patients will respond better to certain treatments

24 Jan 2013

Salk Institute to establish Helmsley Center for Genomic Medicine

The Salk Institute for Biological Studies has received a $42 million gift-the largest in the Institute's history-to establish the Helmsley Center for Genomic Medicine (HCGM), a research center dedicated to decoding the common genetic factors underlying many complex chronic human diseases.

23 Jan 2013

Gene therapy does not slow or halt progression of cell loss in Leber congenital amaurosis

Independent clinical trials, including one conducted at the Scheie Eye Institute at the Perelman School of Medicine, have reported safety and efficacy for Leber congenital amaurosis (LCA), a congenital form of blindness caused by mutations in a gene (RPE65) required for recycling vitamin A in the retina. Inherited retinal degenerative diseases were previously considered untreatable and incurable.

23 Jan 2013

Novel way to engineer specialized immune cells targeted by the AIDS virus

Researchers at the Stanford University School of Medicine have found a novel way to engineer key cells of the immune system so they remain resistant to infection with HIV, the virus that causes AIDS.

23 Jan 2013

Research on dystrophin gene sequence could lead to treatments for Duchenne muscular dystrophy

Muscular dystrophy is caused by the largest human gene, a complex chemical leviathan that has confounded scientists for decades. Research conducted at the University of Missouri and described this month in the Proceedings of the National Academy of Sciences has identified significant sections of the gene that could provide hope to young patients and families.

23 Jan 2013

Gene therapy improves vision but does not halt progression of cell loss

22 Jan 2013

FDA approves Protein Sciences’ Flublok to prevent seasonal influenza

The US Food and Drug Administration has approved Protein Sciences Corporation's vaccine Flublok for the prevention of seasonal influenza in adults 18 to 49 years of age. Diamyd Medical holds about 8 percent of the shares in Protein Sciences Corporation.

18 Jan 2013

Positive gene therapy results in large mammals of Duchenne muscular dystrophy

Usually, results from a new study help scientists inch their way toward an answer whether they are battling a health problem or are on the verge of a technological breakthrough. Once in a while, those results give them a giant leap forward. In a preliminary study in a canine model of Duchenne muscular dystrophy (DMD), University of Missouri scientists showed exactly such a leap using gene therapy to treat muscular dystrophy.

17 Jan 2013

Study demonstrates ways in which quality measurement from EHRs can be improved

Health care providers and hospitals are being offered up to $27 billion in federal financial incentives to use electronic health records (EHRs) in ways that demonstrably improve the quality of care. The incentives are based, in part, on the ability to electronically report clinical quality measures.

16 Jan 2013

Experimental vaccine elicits antibodies that can protect against Ebola virus infection

Researchers at the National Institutes of Health (NIH) and Oregon Health & Science University (OHSU) have found that an experimental vaccine elicits antibodies that can protect nonhuman primates from Ebola virus infection.

16 Jan 2013

Aggressive breast cancer’s metastasis molecular switch identified

Scientists at Weill Cornell Medical College have discovered the molecular switch that allows aggressive triple negative breast cancer cells to grow the amoeba-like protrusions they need to crawl away from a primary tumor and metastasize throughout the body. Their findings, published in Cancer Cell, suggest a novel approach for developing agents to treat cancer once it has spread.

15 Jan 2013

Bionic devices: an interview with Dr Rylie Green

Bionic devices are implants which replace biological functions which have been lost due to nerve damage. They use electrical signals to stimulate the remaining nerve cells following disease or injury. Although the term bionic was only coined in the late 1950s, the earliest bionic devices were cardiac pacemakers, developed in the early 1900s. However the first commercial implantable units were not available until the 1950s.

14 Jan 2013

Blocking TRIP-Br2 gene protects against obesity and insulin resistance

Scientists observed that blocking the expression of the gene TRIP-Br2 in mice protects them against obesity and insulin resistance. The study shows that the gene modulates fat storage by regulating energy expenditure and lipolysis, the process which transforms fat into lipids for the body's energy consumption.

11 Jan 2013

Saliva gland test shows promise for Parkinson's

New research suggests that testing a portion of a person's saliva gland may be a way to diagnose Parkinson's disease.

11 Jan 2013

SLU researchers to test new screening method for MPS disorders among infants

Researchers at Saint Louis University have received a five-year, $2.5 million National Institutes of Health grant to test a new method of screening newborns for a group of rare, but serious genetic disorders that affect approximately 200 babies born in the U.S., each year.

11 Jan 2013