Efficacy of a Bicistronic Vector for Correction of Sandhoff Disease in a Mouse Model

Published 2018 View Full Article

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Title
Efficacy of a Bicistronic Vector for Correction of Sandhoff Disease in a Mouse Model
Authors
Keywords
Sandhoff disease, Tay-Sachs disease, AAV9, gene therapy, hexosaminidase
Journal
Molecular Therapy-Methods & Clinical Development
Volume 12, Issue -, Pages 47-57
Publisher
Elsevier BV
Online
2018-10-26
DOI
10.1016/j.omtm.2018.10.011

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