Ex-vivo modification of autologous CD34+ HSPCs using a CD11b-directed lentiviral vector encoding ApoEII-tagged human IDS leads to supraphysiological enzyme activity and biochemical correction of neuronopathic MPS II patients

Molecular Genetics and Metabolism 2026

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Authors: Simon Allan Jones, Nathan Jeffreys, Kelly E. Watts, Stewart Rust, Heather J. Church, Claire Booth, Karen F. Buckland, Rebecca J. Holley, Brian Bigger, Robert Francis Wynn
DOI: 10.1016/j.ymgme.2025.109489
Cited by: 0

Fields of Study & Subjects

Lysosomal Storage Disorders Research Virus-based gene therapy research Neurogenetic and Muscular Disorders Research

Publication details

Journal / source
Molecular Genetics and Metabolism
Volume
147
Issue
2
Pages
109489–109489
Publication date
1 February 2026
Language
EN