Gene Therapy for Oculopharyngeal Muscular Dystrophy
Human Trials are Near
The team hopes to begin conducting human trials as soon as next year based on the success rates they’ve gotten with mouse models as well as the now simplified vector model. The beauty of this single vector gene therapy system is that it can be used to treat and possibly eliminate other forms of MD as well, but the technology has applications across the medical field as a more simple vector for gene therapies for different disease and disorders.
