Lentiviral stem cell gene therapy of neuronopathic Gaucher disease (GD3) achieves prolonged enzyme delivery, substrate reduction and stabilisation of neurologic and somatic disease manifestations

Molecular Genetics and Metabolism 2026

View at publisher
Authors: Aimée Donald, Alexander A. Broomfield, Jane Potter, Kelly E. Watts, Stewart Rust, James Cooper, Heather J. Church, Simon Allan Jones, Robert Francis Wynn
DOI: 10.1016/j.ymgme.2025.109391
Cited by: 0

Fields of Study & Subjects

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

Publication details

Journal / source
Molecular Genetics and Metabolism
Volume
147
Issue
2
Pages
109391–109391
Publication date
1 February 2026
Language
EN