Gene therapy is linked to better nonmotor outcomes in spinal muscular atrophy type 1 compared to first-line nusinersen, new ...

An experimental gene therapy developed by researchers at UCLA, University College London and Great Ormond Street Hospital has ...

Regeneron Pharmaceuticals, Inc. REGN announced updated data on its investigational gene therapy DB-OTO from the CHORD study.

Despite announcing a broad pivot to siRNA earlier this year, Sarepta is following through with an investigational gene ...

An experimental gene therapy developed by researchers at UCLA, University College London and Great Ormond Street Hospital has ...

New research found that gene therapy for children with a form of severe combined immunodeficiency was successful in 95% of ...

Results from the largest cohort of children who received a gene therapy for a rare immunodeficiency condition have shown the ...

These results are what we hoped for when we first began developing this approach,' one researcher said. View on euronews ...

In a study published in Nature Medicine, Leone and her team reported the first targeted gene therapy to restore myelin, the ...

A new breakthrough in a rare genetic disease which causes children to age rapidly has been discovered using 'longevity genes' ...

Researchers convert type A kidney to type O, potentially reducing wait times and saving lives. Experimental gene therapy ...

Stevie Ringel is the founder of the Kizuna Foundation, an organization seeking a treatment for a genetic retinal mutation ...