Cell and Gene Therapy News

At 4 years old, Eliot was the first patient at CHLA to receive Elevidys, a groundbreaking new gene therapy for Duchenne muscular dystrophy. The treatment has been a game changer for his health.

An international expert at CHLA talks about the state of hemophilia gene therapy—and what’s on the horizon.

A national leader in pediatric gene therapy, CHLA has treated more patients with the sight-saving Luxturna than any other center in the country.

A CHLA expert shares her experience of administering this gene therapy for Duchenne—and why comprehensive care is critical for patients.

Gene therapy holds tremendous potential for children, but many promising therapies are shelved. Here’s what a new model could look like.

How next-generation gene therapies target the underlying causes of genetic diseases.