Safety of AAV gene therapy programs for neurological indications – Learning from programs in non-lysosomal disorders

Molecular Genetics and Metabolism 2026

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Authors: Aimée Donald, Rafael Escandon, Barry Byrne, Simon Allan Jones
DOI: 10.1016/j.ymgme.2025.109393
Cited by: 0

Fields of Study & Subjects

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

Publication details

Journal / source
Molecular Genetics and Metabolism
Volume
147
Issue
2
Pages
109393–109393
Publication date
1 February 2026
Language
EN