AAV Therapy: A Successful One-Patient SPG50 Gene Therapy Trial

News Article Author(s): An adeno-associated virus (AAV) gene therapy for hereditary spastic paraplegia type 50 (SPG50)—funded by a young patient’s family—gives hope not only to a 4-year-old, but to researchers working on similar projects for other rare diseases. In hereditary spastic paraplegia type 50 (SPG50)—an ultrarare form of neurodevelopmental/neurological disorder described as ideal for gene therapy—Canadian researchers successfully created and completed a phase 1 gene replacement therapy trial for a single patient. They presented their results in Nature Medicine, also …