In LAMA2-related muscular dystrophy, weakened extracellular matrix (yellow) leads to muscle fiber degeneration and the infiltration of immune cells (red) into the tissue. Researchers at the University ...

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.

A blood stem cell gene therapy co-developed by UCLA’s Dr. Donald Kohn restored immune function in 59 of 62 children with ADA-SCID, a rare and fatal immune disorder, with no serious complications ...

For the first time, scientists have found a way to dramatically slow Huntington’s disease, an inherited brain disorder that has long been considered untreatable. A groundbreaking gene therapy called ...

KaDee Troop is the mother of seven adopted children, four of whom have a rare genetic disorder that causes their skin to blister and tear at the slightest touch. Wounds heal slowly — if at all — and ...

UF scientist develops safer gene therapy restoring vision in children with rare inherited eye diseases; pivotal trials begin ...

Five-year clinical study results show this revolutionary one-time treatment allowed 94% of patients to stop routine factor IX prophylaxis "I'm just very blessed to be in the right spot at the right ...

Please provide your email address to receive an email when new articles are posted on . This is the first biologics license application submitted for a gene-agnostic retinal disease gene therapy.

This article is part of Nature Outlook: Skin, an editorially independent supplement produced with financial support from LEO Pharma. About this content. KaDee Troop is the mother of seven adopted ...