At a Food and Drug Administration cell and gene therapy (CGT) roundtable last June, most speakers pushed for the agency to ...

A central nervous system (CNS) tumor has prompted the FDA to place clinical holds on two Regenxbio gene therapies, including ...

An international workshop of scientists, regulators, and industry experts led by Prof Ivana Barbaric convened to address the risks of genetic mutations in stem cell therapy. Subscribe to our ...

Gene therapy approaches include gene replacement, suppression, and editing, each matched to specific genetic mechanisms in hereditary hearing loss. Preclinical studies in rodent models show promise, ...

Ryuji Morizane, MD, PhD, of the Department of Medicine at Massachusetts General Hospital, is the senior/corresponding author of a new paper published in Signal Transduction and Targeted Therapy, "AAV ...

Forbes contributors publish independent expert analyses and insights. A patient in a late-stage gene editing trial for a rare heart condition died from fatal liver complications after receiving an ...

Duchenne muscular dystrophy (DMD) is a neuromuscular disorder that results from mutations in the DMD gene. Gene therapies for DMD change genetic material in a person’s body to treat this condition.