Hematopoietic stem cell gene therapy for mucopolysaccharidosis type IIIC

Molecular Genetics and Metabolism 2026

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Authors: Rafael A. Badell‐Grau, Frankie Son, Melissa Rubalcava Sandoval, Kevin Eric Thai, Kasra Pakravesh, Gregoire Agnus, Greyson Tong, Quinn Brown, Alexis N. Corl, Tara Shammas, Veenita Khare, Anusha Sivakumar
+2 more Johannes C. M. Schlachetzki, Stéphanie Cherqui
DOI: 10.1016/j.ymgme.2025.109323
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
109323–109323
Publication date
1 February 2026
Language
EN