Improving Single Injection CSF Delivery of AAV9-mediated Gene Therapy for SMA: A Dose–response Study in Mice and Nonhuman Primates

Title
Improving Single Injection CSF Delivery of AAV9-mediated Gene Therapy for SMA: A Dose–response Study in Mice and Nonhuman Primates
Authors
Keywords
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Journal
MOLECULAR THERAPY
Volume 23, Issue 3, Pages 477-487
Publisher
Elsevier BV
Online
2014-10-31
DOI
10.1038/mt.2014.210

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