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An empowered, clinically viable hematopoietic stem cell gene therapy for the treatment of multisystemic mucopolysaccharidosis type II

2024

Das S et Al.

Mol Ther 32, 619-636.

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Genethon is a member of the Biotherapies Institute for Rare Diseases that was created by AFM-Telethon

Biotherapies Institute

Institute of Myology

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