Improving Single Injection CSF Delivery of AAV9-mediated Gene Therapy for SMA: A Dose–response Study in Mice and Nonhuman Primates
Titel:
Improving Single Injection CSF Delivery of AAV9-mediated Gene Therapy for SMA: A Dose–response Study in Mice and Nonhuman Primates
Auteur:
Meyer, Kathrin Ferraiuolo, Laura Schmelzer, Leah Braun, Lyndsey McGovern, Vicki Likhite, Shibi Michels, Olivia Govoni, Alessandra Fitzgerald, Julie Morales, Pablo Foust, Kevin D Mendell, Jerry R Burghes, Arthur H M Kaspar, Brian K