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

Researchers have developed a simple model system that can be used to break down fibrils -- the cause of numerous disorders including Alzheimer's and Parkinson's disease -- into their constituent ...

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