Novel intravenous AAV gene therapy for mucopolysaccharidosis type IIIA and IIIB in mouse and canine model of the pathology – Towards clinical translation

Molecular Genetics and Metabolism 2026

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Authors: Françoise Piguet, Rafael Alonso, Elena Gaia Banchi, Thibaut Marais, Nissrine Ballout, Johan Deniaud, Sylvie Jacquot, David El Hajj, Corinne Jounier, Stéphane Madec, David Augereau, Marion Fusellier
+4 more D. Bencharif, Françoise Roux, Marie-Anne Colle, Jérôme Ausseil
DOI: 10.1016/j.ymgme.2025.109582
Cited by: 0

Fields of Study & Subjects

Lysosomal Storage Disorders Research Virus-based gene therapy research RNA Interference and Gene Delivery

Publication details

Journal / source
Molecular Genetics and Metabolism
Volume
147
Issue
2
Pages
109582–109582
Publication date
1 February 2026
Language
EN