Therapeutic benefits of hematopoietic stem cell gene therapy using optimized bicistronic lentiviral vectors in mouse models of GM2 gangliosidosis

Molecular Genetics and Metabolism 2026

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Authors: Angela Gritti, Simone Borsani, Francesco Morena, Davide Sala, Marco Patrone, Massimo Degano, Sabata Martino, Francesca Ornaghi
DOI: 10.1016/j.ymgme.2025.109447
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
109447–109447
Publication date
1 February 2026
Language
EN