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Transplantation of Wild-Type Hematopoietic Stem and Progenitor Cells Improves Disease Phenotypes in a Mucopolysaccharidosis IIIC Mouse Model.

Cell transplantation - 1 Jan 2025

Badell-Grau Rafael A, Pakravesh Kasra, Thai Kevin Eric, Son Frankie, Chen Rola, Rainaldi Joseph, Duong Kalvin, Losay Pauline, Sivakumar Anusha, Khare Veenita, Corl Alexis N, Pithia Rushil, Tran Christine, Esko Jefferey D, Cherqui Stephanie

Abstract excerpt

Mucopolysaccharidosis type IIIC (MPS IIIC) is a severe neurodegenerative lysosomal storage disease caused by the loss-of-function of the lysosomal transmembrane protein acetyl-CoA: heparan-α-glucosamine N-acetyltransferase. MPS IIIC is characterized by the accumulation of the glycosaminoglycan (GAG) heparan sulfate. There is no treatment for this disease. We generated a new MPS IIIC mouse model and confirmed...

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Identifiers

PMID: 40126917
DOI: 10.1177/09636897251323966
PMCID: PMC11938874

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