Gene Therapy

Credit: Walter and Eliza Hall Institute, Australia New funding is enabling Walter and Eliza Hall Institute researchers to develop new approaches to potentially help people with Prader-Willi syndrome, a devastating and incurable…

Credit: Trotman lab/CSHL Cold Spring Harbor, NY -- An estimated 450,000 Americans are diagnosed each year with a cancer that's driven by MYC, an oncogene that hijacks growth and metabolism in cancer cells. MYC has a storied…

A research team receives the 2019 Human Growth Award for a clinical trial of growth hormone treatment provided to patients with short stature due to aggrecan deficiencyCredit: Children's National Health System On Sunday, April 28, a team…

Credit: Photo by Fred Zwicky CHAMPAIGN, Ill. -- A fortuitous conversation between two University of Illinois scientists has opened a new line of communication between biomedical researchers and the tissues they study. The new findings,…

Scientists find the answer in a single gene mutation AURORA, Colo. (May 6, 2019) - A new immunodeficiency disease caused by a novel genetic mutation has been identified by researchers at the University of Colorado Anschutz Medical Campus…

Widely reported case experiment is unlikely to provide intended protection, with unknown risks The first reported instance of germline gene editing in humans was bad science as well as bad ethics, according to a commentary publishing April…

The public announcement last fall from China regarding gene editing on human embryos, conducted without the benefit of scientific review or ethical debate, was met with worldwide disdain. It also has raised global concerns that more…

CORVALLIS, Ore. - A pharmaceutical sciences researcher at Oregon State University has received a five-year, $3.3 million grant from the National Institutes of Health to develop a way for cystic fibrosis patients to get molecular treatment…

Credit: Scott Wiseman for Scripps Research JUPITER, Fla. - April 29, 2019 - A new study challenges the presumption that people born with developmental brain disorders such as severe autism will benefit from medical interventions only if…

Axon 'insulating tape' suffers from gene deletion that causes social impairment associated with rare disorder, Tel Aviv University researchers say A new Tel Aviv University study finds that gene deletion or deficiency in neurons is…