By adding a microRNA molecule to ALS models, Tel Aviv University, Ben-Gurion University and Weizmann Institute researchers ...

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

For more than a decade, the strongest AIDS drugs could not fully control Matt Chappell's HIV infection. Now his body controls it by itself, and researchers are trying to perfect the gene editing that ...

The U.S. Food and Drug Administration is tightening restrictions on a gene therapy used to treat Duchenne muscular dystrophy ...

The approach is “very exciting,” but one expert says a lot more research is needed, especially around safety, before clinical ...

MeiraGTx is cashing in on a gene therapy that restored vision to 11 children who were born legally blind, licensing the ...

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.

Fred Hutch bioengineer Dr. Matthias Stephan is working to develop a special foam that could help make gene therapy for blood ...

The FDA has restricted the number of patients who can receive the Cambridge drugmaker's gene therapy for Duchenne muscular ...