Scientists at St. Jude Children's Research Hospital and Northwestern University identified a previously unknown treatment ...

Adeno-associated virus (AAV)–mediated gene therapy was one of the focal points of this year's Muscular Dystrophy Association conference. Here, Barry Byrne, MD, PhD, University of Florida, speaks to ...

Researchers report early but encouraging results on an experimental gene-editing treatment for high cholesterol.

Your heart will not just give you fear and uncertainty after a heart attack; it can leave scar tissue that stiffens your ...

The search for gene therapies to treat rheumatoid arthritis (RA) has moved in fits and starts over the past two decades, with trials starting and stopping, and their focus shifting to osteoarthritis ...

As a new €2 million gene therapy research project funded by APC and Research Ireland is announced, we speak with the project lead Prof Niall Barron about its potential and how Ireland is positioning ...

Boston biotech Beam Therapeutics has announced initial data from a phase 1/2 trial of its gene therapy for a genetic lung and liver disease, with no serious adverse events reported among the nine ...

Viral vectors dominate gene therapy, with lentivirus, adenovirus, and AAV being key players, each with unique advantages and limitations. Non-viral vectors, such as lipid nanoparticles and GalNAc, ...

Researchers in the lab of Hans-Peter Kiem, MD, PhD, at Fred Hutch Cancer Center have devised a method that could one day treat genetic hematologic disorders by correcting how the body makes blood ...

The agency is establishing a network of care centers to expand access to regenerative medicine beyond regions served by major ...