In a first for adults in Singapore, scientists are conducting gene editing trials on heart patients to correct defects at ...

The Boston biotech founded by Harvard professor David Sinclair is testing whether cellular reprogramming can turn back the ...

Last May, people were thrilled to learn that a 7-month-old baby who became the first in the world to receive a personalized ...

In 1962, a pediatric neurology resident at Columbia's Neurological Institute and his colleagues in the College of Physicians ...

In honor of World Blindness Awareness Month, "GMA" is spotlighting a breakthrough gene therapy that's helping some blind ...

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

In the spring of 2024, the US Food and Drug Administration (FDA) approved fidanacogene elaparvovec (Beqvez), only the second gene therapy product for hemophilia B. Just a few months later, the Pfizer ...

Oncologists' experience with CGT has increased, with more patients treated and earlier lines of therapy being utilized.

Duke Eye Center has become the first academic medical center in the country to implant a new cell-based gene therapy for a rare eye disease called MacTel. This procedure offers hope to patients like ...

With Eli Lilly's recent acquisition of Verve Therapeutics and its PCSK9 candidate, delve into what the target represents.

The trial was small — just 12 children were treated in one or both ears — but it offers important lessons for researchers about the potential of Regeneron’s 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.