Scientists at St. Jude Children's Research Hospital and Northwestern University identified a previously unknown treatment opportunity for sickle cell disease and β-thalassemia. The discovery, ...
X-linked retinoschisis gene replacement therapy, aiming to improve retinal architecture and visual function in young male ...
Moorfields Eye Hospital in London made international headlines last week. Doctors announced they had saved the sight of four young children suffering from a rare genetic condition that rapidly causes ...
Parkinson disease (PD) is a debilitating, progressive neurological disorder caused by degeneration of dopaminergic neurons in the basal ganglia of the brain. Current treatments focus on boosting ...
Today, after receiving a gene therapy developed at the University of California San Diego, Natalie is thriving at 34 years ...
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 ...
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 ...
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