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Human Gene Therapy|October 13, 2022
Phenotypic Correction of Murine Mucopolysaccharidosis Type II by Engraftment of Ex Vivo Lentiviral Vector-Transduced Hematopoietic Stem and Progenitor CellsMiles C Smith, Lalitha R Belur, Andrea D Karlen, et al.
Molecular Therapy. Methods & Clinical Development|December 17, 2024
Intrathecal or intravenous AAV9-IDUA/RGX-111 at minimal effective dose prevents cardiac, skeletal and neurologic manifestations of murine MPS ILalitha R Belur, Avery K Huber, Hillary Mantone, et al.
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