Development of a novel systemic AAV gene therapy for neuronopathic Gaucher disease

Molecular Genetics and Metabolism 2026

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Authors: Tae Un Han, Pamela Head, Bahafta A. Berhe, Charles P. Venditti, Ellen Sidransky

DOI: 10.1016/j.ymgme.2025.109455

Cited by: 0

Fields of Study & Subjects

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