A New AAV10-U7-Mediated Gene Therapy Prolongs Survival and Restores Function in an ALS Mouse Model

Title
A New AAV10-U7-Mediated Gene Therapy Prolongs Survival and Restores Function in an ALS Mouse Model
Authors
Keywords
ALS, AAV, exon skipping, gene therapy, hSOD1
Journal
MOLECULAR THERAPY
Volume 25, Issue 9, Pages 2038-2052
Publisher
Elsevier BV
Online
2017-06-27
DOI
10.1016/j.ymthe.2017.05.017

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