Phenotypic Correction of Murine Mucopolysaccharidosis Type II by Engraftment of <i>Ex Vivo</i> Lentiviral Vector-Transduced Hematopoietic Stem and Progenitor Cells.

Hum Gene Ther · 2022 · PMC9808798 · PMID 36226412

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In conclusion, transplantation of MNDU3-IDS HSPCs into MPS II mice showed a trend toward normalization of neurobehavioral outcomes, indicating the potential for prevention of neurocognitive deficiency.

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