Researchers developed a dual-AAV gene therapy for Usher syndrome type 1F, restoring hearing and balance in mice. The approach ...

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

Delivering very large genes to cells can pose a serious challenge, so scientists developed a new method. | Genetics And ...

Single-Dose Gene Therapy Is Potentially Life-Changing for ... have used gene editing to restore hearing in adult mice with a type of inherited hearing loss. They showed that shutting down a ...

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.

University of Wisconsin–Madison researchers targeting a group of hereditary neurodegenerative diseases have found success ...

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

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

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