Gene Therapy

A new study analyzes 30 years of patient data and details the clinical course of 184 individuals with genetically diverse forms of maple syrup urine disease STRASBURG, PA- A new study analyzes 30 years of patient data and details the…

We live in an increasingly complex world when it comes to chemicals. The number of new chemicals has increased from 20 million in 2002 to 156 million last year. Many of these are ubiquitous in the world around us because of their…

Identification of GPR108 as 'lock' for adeno-associated virus vector (AAV) is crucial to this emerging novel class of genetic medicine and may provide framework for developing more targeted gene therapiesCredit: Pierce Harman …

Scientists find unexpected connection between 2 main forms of AMDCredit: Dan Addison | UVA Communications Researchers have successfully treated age-related macular degeneration (AMD) in mice after finding an unexpected link between the two…

A team of researchers at CHU Sainte-Justine and Université de Montréal has shed light on the mechanisms that underlie a rare genetic condition by creating the first cellular model of the disease. The study's findings were published today…

UNIGE researchers have discovered a new gene that causes blindness and cardiomyopathy; they have also managed to halt the progression of eye disease and treat cardiac disease by administering a food supplementCredit: © UNIGE Our genome…

Research identifies mechanism of synaptic damage in certain neurodegenerative disordersCredit: UMass Medical School New research by Dorothy P. Schafer, PhD, at the University of Massachusetts Medical School, reveals the molecular…

April 22-26, 2020 in DC Metro area Reporters are invited to attend The Allied Genetics Conference (TAGC), to be held April 22-26, 2020 in National Harbor, Maryland, just 30 minutes from downtown Washington DC. TAGC 2020 will feature the…

Findings from a collaborative clinical trial have generated key insights into how targeted therapy should be used to treat leukemia driven by the Philadelphia chromosomeCredit: St. Jude Children's Research Hospital Researchers from St.…

The brain is a sort of fortress, equipped with barriers designed to keep out dangerous pathogens. But protection comes at a cost: These barriers interfere with the immune system when faced with dire threats such glioblastoma, a deadly…