PS gene-editing system corrects Sandhoff disease with a brain-penetrant HEXO-APOE enzyme

Molecular Genetics and Metabolism 2026

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Authors: Jeffrey Gaynes, Li Ou, Michael J. Przybilla, Jeanine R. Jarnes, Chester B. Whitley

DOI: 10.1016/j.ymgme.2025.109421

Cited by: 0

Fields of Study & Subjects

Lysosomal Storage Disorders Research Virus-based gene therapy research Glycogen Storage Diseases and Myoclonus