Sustained supraphysiological alpha-L-iduronidase (IDUA) activity, reduction of glycosaminoglycans (GAGs), and clinical benefits at 5 years post-treatment with OTL-203, an autologous hematopoietic stem cell gene therapy (HSC-GT), in patients with mucopolysaccharidosis type I (MPS IH) Hurler syndrome

Molecular Genetics and Metabolism 2026

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Authors: Maria Ester Bernardo, Francesca Tucci, Giulia Consiglieri, Francesca Fumagalli, Maurizio De Pellegrin, Francesca Ciotti, Marina Sarzana, Vasileios Nikolaou, Jean Brooks, Marianna Lalla, Muska N. Yarzi, Su Syonmez
+2 more Laura Campbell, Alessandro Aiuti
DOI: 10.1016/j.ymgme.2025.109333
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
109333–109333
Publication date
1 February 2026
Language
EN