Neonatal brain-directed gene therapy rescues a mouse model of neurodegenerative CLN6 Batten disease.
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.
Kleine Holthaus SM, et al. Among authors: klaska ip.
Hum Mol Genet. 2019 Dec 1;28(23):3867-3879. doi: 10.1093/hmg/ddz210.
Hum Mol Genet. 2019.
PMID: 31807779