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Acta Neuropathologica Communications|November 2, 2018
Satellite cells fail to contribute to muscle repair but are functional in Pompe disease (glycogenosis type II)Lydie Lagalice, Julien Pichon, Eliot Gougeon, et al.
Acta Neuropathologica Communications|June 24, 2026
Accumulation of membrane repair-associated proteins and mature myostatin are novel markers of muscle pathophysiology in Pompe diseaseCandice Babarit, Sabrina Jagot, Cindy Schleder, et al.
Human Gene Therapy. Clinical Development|March 12, 2015
Intracerebral Gene Therapy Using AAVrh.10-hARSA Recombinant Vector to Treat Patients with Early-Onset Forms of Metachromatic Leukodystrophy: Preclinical Feasibility and Safety Assessments in Nonhuman PrimatesMichel Zerah, Françoise Piguet, Marie-Anne Colle, et al.
Molecular Therapy. Methods & Clinical Development|May 2, 2020
Intra-CSF AAV9 and AAVrh10 Administration in Nonhuman Primates: Promising Routes and Vectors for Which Neurological Diseases?Karim Bey, Johan Deniaud, Laurence Dubreil, et al.
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