Neonatal brain-directed gene therapy rescues a mouse model of neurodegenerative CLN6 Batten disease

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Authors: kleine Holthaus SM, Herranz-Martin S, Massaro G, Aristorena M, Hoke J, Hughes MP, Maswood R, Semenyuk O, Basche M, Shah AZ, Klaska IP, Smith AJ, Mole SE, Rahim AA, Ali RR

Journal publisher: biorxiv

Published year: 2019

DOI identifier: 10.1101/673848