Gene therapy for rare disease involves gene transfer techniques that alter or modify the expression of a gene or its activity to cure genetic disorders. Gene therapy is emerging as a viable treatment option for various rare genetic diseases, such as hemophilia, hearing loss, retinitis pigmentosa, Gaucher's disease, and Sanfilippo syndrome, for which conventional treatment options are not available or are highly inadequate. The method is advantageous as it can potentially provide a functional ... Read more
