Improving Single Injection CSF Delivery of AAV9-mediated Gene Therapy for SMA: A Dose–response Study in Mice and Nonhuman Primates
Meyer, Kathrin, Ferraiuolo, Laura, Schmelzer, Leah, Braun, Lyndsey, McGovern, Vicki, Likhite, Shibi, Michels, Olivia, Govoni, Alessandra, Fitzgerald, Julie, Morales, Pablo, Foust, Kevin D, Mendell, JeVolume:
23
Langue:
english
Journal:
Molecular Therapy
DOI:
10.1038/mt.2014.210
Date:
March, 2015
Fichier:
PDF, 4.72 MB
english, 2015
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