Sustained biochemical correction and improved neurological outcomes at 36-months post hematopoietic stem cell gene therapy for Sanfilippo syndrome

Molecular Genetics and Metabolism 2026

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Authors: Brian Bigger, Jane Louise Kinsella, Jane Potter, Karen F. Buckland, Heather J. Church, Rachel Searle, Stuart M. Ellison, Yuko Ishikawa Learmonth, Rebecca J. Holley, Claire Booth, Adrian James Thrasher, Stewart Rust
+2 more Simon Allan Jones, Robert Francis Wynn
DOI: 10.1016/j.ymgme.2025.109344
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
109344–109344
Publication date
1 February 2026
Language
EN