Long-term follow-up shows 95% success rate, no serious complications in largest ADA-SCID gene therapy study to date ...

A one-time gene therapy using a patient’s own stem cells has effectively cured a deadly immune disorder in 95% of treated ...

“Beyond UniQure, the pipeline includes highly promising approaches that will ideally move the goal from merely managing ...

For children born with severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID), an investigational ...

Results from the largest cohort of children who received a gene therapy for a rare immunodeficiency condition have shown the ...

Huntington’s disease has been successfully treated for the first time using a gene therapy, which may be available in the US as soon as next year ...

Gene therapy is becoming a powerful way to treat challenging diseases that don’t respond to traditional treatments, and researchers now report the first success in modifying genes to slow Huntington’s ...

New research found that gene therapy for children with a form of severe combined immunodeficiency was successful in 95% of ...

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 ...

Editor’s note: On June 3, 2025, KJ Muldoon was discharged from the hospital and is now at home with his family. When a baby born in Philadelphia was announced as the first person to get a gene therapy ...

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.

Four patients with infantile-onset Pompe’s disease received a single intravenous injection of an adeno-associated virus serotype 9 vector carrying codon-optimized complementary DNA encoding human acid ...