The promise of gene therapy looms large for families dealing with rare, genetic disorders. Such treatments offer the possibility of one-time cures. But families and researchers worry such therapies will …
Robin Alderman faces an agonizing reality: Gene therapy might cure her son Camden’s rare, inherited immune deficiency. But it’s not available to him. In 2022, London-based Orchard Therapeutics stopped investing …
MBALE, Uganda — Barbara Nabulo was one of three girls in her family. But when a sister died, her mother wailed at the funeral that she was left with just …
WASHINGTON DC: A new study in mice suggests that replacing a dysfunctional gene may improve survival in some people with arrhythmogenic right ventricular cardiomyopathy (ARVC), a rare inherited disorder in …
To get the new genetic material into cells, they engineered harmless viruses to carry it. Doctors carefully injected a tiny amount of liquid containing the viruses into a part of …
Gautam Dongre’s two children in India and Pascazia Mazeze’s son in Tanzania live with an inherited blood disorder that turns blood cells into instruments of pain. New gene therapies promise …
Our B cells help prevent us from getting sick. Their job is to make antibodies, immune system proteins that fight off viruses and other foreign invaders. And they make a …
Gautam Dongre’s two children in India and Pascazia Mazeze’s son in Tanzania live with an inherited blood disorder that turns blood cells into instruments of pain. Now that new gene …
New Delhi: The US Food and Drug Administration (USFDA) last week approved two breakthrough gene therapies — Casgevy by Vertex Pharmaceuticals and CRISPR Therapeutics, and Lyfgenia by Bluebird Bio — for sickle …
The U.S. Food and Drug Administration (FDA) on Friday approved a pair of gene therapies for sickle cell disease, including the first treatment based on the breakthrough CRISPR gene editing …