StitchR makes it possible to deliver larger payloads with current vectors, expanding the list of diseases targetable by gene therapies.
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 a big problem: size.
An international research team has uncovered a new mechanism crucial to the production of cellular proteins. When this ...