Efficacy of AAV-mediated gene therapy in a sheep model of CLN1 disease

Molecular Genetics and Metabolism 2026

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Authors: Grace Robinson Kick, Samantha L. Eaton, Sophie H. Wang, Elizabeth M. Eultgen, Ewa Ziółkowska, Gerard Thompson, Mark Gray, Mark S. Sands, Beverly L. Davidson, Thomas M. Wishart, Jonathan D. Cooper
DOI: 10.1016/j.ymgme.2025.109499
Cited by: 0

Fields of Study & Subjects

Lysosomal Storage Disorders Research Virus-based gene therapy research RNA Interference and Gene Delivery

Publication details

Journal / source
Molecular Genetics and Metabolism
Volume
147
Issue
2
Pages
109499–109499
Publication date
1 February 2026
Language
EN