Improving Single Injection CSF Delivery of AAV9-mediated Gene Therapy for SMA: A Dose–response Study in Mice and Nonhuman Primates
Kathrin Meyer(Nationwide Children's Hospital), Laura Ferraiuolo(Nationwide Children's Hospital), Leah Schmelzer(Nationwide Children's Hospital), Lyndsey Braun(Nationwide Children's Hospital), Vicki L. McGovern(The Ohio State University Wexner Medical Center), Shibi Likhite(Nationwide Children's Hospital), Olivia Michels(Nationwide Children's Hospital), Alessandra Govoni(Nationwide Children's Hospital), Julie C. Fitzgerald(The Ohio State University), Pablo Morales, Kevin D. Foust(The Ohio State University), Jerry R. Mendell(Nationwide Children's Hospital), Arthur H.M. Burghes(The Ohio State University Wexner Medical Center), Brian K. Kaspar(Nationwide Children's Hospital)
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