A Novel Small NPC1 Promoter Enhances AAV-Mediated Gene Therapy in Mouse Models of Niemann–Pick Type C1 Disease

por Hughes, Michael Paul, Nelvagal, Hemanth Ramesh, Coombe-Tennant, Oliver, Smith, Dave, Smith, Claire, Massaro, Giulia, Poupon-Bejuit, Laura, Platt, Frances Mary, Rahim, Ahad Abdul
Publicado 2023