Gene Therapy for Lysosomal Storage Disorders: Ongoing Studies and Clinical Development

por Massaro, Giulia, Geard, Amy F., Liu, Wenfei, Coombe-Tennant, Oliver, Waddington, Simon N., Baruteau, Julien, Gissen, Paul, Rahim, Ahad A.
Publicado 2021

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