Musculoskeletal

Mouse studies suggest combining knockdown of mutant PABPN1 with expression of normal PABPN1 could help treat oculopharyngeal muscular dystrophy (OPMD). In a mouse model of OPMD, intramuscular injection of shRNA targeting mutant PABPN1 plus an adeno-associated viral (AAV) vector encoding normal PABPN1 decreased muscle fibrosis, increased myofiber size and muscle function compared with intramuscular vehicle. Next steps by Benitec Biopharma Ltd. include combining the two parts of the therapy into a single vector and planning for Phase I/IIa testing...