A single shot of gene therapy restored hearing in people born deaf, with most showing improvement within weeks and some ...

Case Western Reserve University chemist Divita Mathur was awarded a National Science Foundation (NSF) Faculty Early Career ...

In a historic medical breakthrough, a three-year-old child has become the youngest patient ever to receive a revolutionary ...

Gabriella Ngang, of Farmington Hills, is one of three in Michigan to take part in a clinical trial of a new sickle cell ...

Boosting Klotho protein levels in mice improves physical and cognitive aging, offering potential for future longevity ...

The pivotal trial for Neurogene's Rett syndrome gene therapy makes use of baseline controls and a rigorous endpoint that ...

New open label data in Becker demonstrated sustained disease stabilization up to three years, reinforcing prior clinical findings – – Ongoing pivotal trial and FDA Type C meeting provide clear path to ...

Researchers have developed a gene therapy that significantly slowed motor function loss in preclinical models of amyotrophic lateral sclerosis (ALS), offering new hope for treating the devastating ...

Alsheebani is one of the first adults in the world, and the first adult at the medical system, to be effectively cured of sickle cell disease using gene therapy. "The doctors kept assuring me that it ...

KJ Muldoon, a 10-month-old baby who sparked nationwide headlines after receiving a first-of-its kind gene-editing treatment, was released from the hospital this week. KJ has spent the majority of ...

The gene therapy trial was expected to complete dosing in the middle of this year. With the clinical hold in place, Shah said the timeline is uncertain. The study’s targeted enrollment was 12 ...

Opus Genetics announces upcoming presentations on gene therapy research for inherited retinal diseases at May 2025 conferences. Quiver AI Summary Opus Genetics, Inc., a clinical-stage ...