A new study has brought scientists one step further in the direction of developing a cure for a brutal group of rare brain ...

A one-time gene therapy using a patient’s own stem cells has effectively cured a deadly immune disorder in 95% of treated ...

Because the new method can replace a large stretch of defective DNA with a healthy sequence, the same retron-based package can fix any combination of mutations within that stretch of DNA without ...

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

Fractyl Health announced a first-in-human trial for RJVA-001, targeting T2D and obesity, pending regulatory approval by 2026. Fractyl Health, Inc. has announced the submission of the first module of ...

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

The goal of gene therapy is to permanently cure hereditary diseases. One of the most promising technologies for this is the ...

Millions of years ago, humans' ancestors lost the function of a specific gene — but switching that gene back on could help protect people from gout, a new experimental study suggests. Gout is a type ...

New research found that gene therapy for children with "bubble boy disease," or SCID, was successful in 95% of trial ...