METHODS FOR TREATING OCULOPHARYNGEAL MUSCULAR DYSTROPHY (OPMD)

摘要

The present disclosure relates to methods of administering a gene therapy construct comprising RNA interference (RNAi) reagents, such as short hairpin microRNA (shmiR), in combination with PABPN1 replacement reagents, such as polynucleotides which encode functional PABPN1 protein which are not targeted by the RNAi reagents, for treatment of oculopharyngeal muscular dystrophy (OPMD) in individuals suffering from OPMD or which are predisposed thereto. In certain aspects the method comprises direct injection to a subject's pharyngeal muscles.