Researchers at Children's Hospital of Philadelphia (CHOP) announced encouraging results from the first ever gene therapy ...

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 ...

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

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

In India, approximately 4,000 children are born with Spinal Muscular Atrophy (SMA) every year, making it a critical, albeit ...

An experimental vaccine showed promise in treating aggressive breast cancer, while a new gene therapy method advances ...

Duchenne muscular dystrophy is the most common early ... a healthy gene copy back to a patient’s cells to correct genetic diseases, but the vectors used to deliver this information are small ...

A new technology that delivers two halves of a gene separately could enable gene therapy treatments of muscular dystrophies.