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Long-term safety and dose escalation of intracerebroventricular CLN5 gene therapy in sheep supports clinical translation for CLN5 Batten disease.
Mitchell NL, Murray SJ, Wellby MP, Barrell GK, Russell KN, Deane AR, Wynyard JR, Palmer MJ, Pulickan A, Prendergast PM, Casy W, Gray SJ, Palmer DN. Mitchell NL, et al. Among authors: deane ar. Front Genet. 2023 Aug 8;14:1212228. doi: 10.3389/fgene.2023.1212228. eCollection 2023. Front Genet. 2023. PMID: 37614821 Free PMC article.