Harvard Medical School researchers have taken another decisive step in their efforts to develop a gene therapy for people ...

StitchR, a new gene therapy technique, delivers large genes in two parts to treat muscular dystrophies by restoring critical proteins in animal models.

Gene therapy can effectively treat various diseases, but for some debilitating conditions like muscular dystrophies there is ...

These preclinical findings could lead ... Single-Dose Gene Therapy Is Potentially Life-Changing for Adults With Hemophilia B Sep. 25, 2024 — Adults with hemophilia B saw their number of bleeding ...

StitchR makes it possible to deliver larger payloads with current vectors, expanding the list of diseases targetable by gene therapies.

An RNA-editing gene therapy has been developed that switches off the key driver of common eye conditions affecting diabetics ...

Gene therapy for some diseases, including Duchenne muscular dystrophy (DMD), can be tricky because the needed gene is often ...

Neurogene shares tumbled 35% last night after it disclosed that a pediatric patient with Rett syndrome experienced a severe ...

Kebilidi is the first approved gene therapy that can be directly administered to the brain. Its OK secures a priority review ...

Gene therapy can effectively treat various diseases, but for some debilitating conditions like muscular dystrophies there is a big problem: size.

The FDA issued its first stamp of approval for a cell or gene therapy back in 2017 to Novartis' Kymriah. | The gene therapy ...

Modifying Treatment for AADC Deficiency in the United States SOLANA BEACH, CA / ACCESSWIRE / November 13, 2024 / ...