Article
Liver-Directed Human Amniotic Epithelial Cell Transplantation Improves Systemic Disease Phenotype in Hurler Syndrome Mouse Model.
Stem cells translational medicine - 1 Jul 2017
Rodriguez Natalie S, Yanuaria Lisa, Parducho Kevin Murphy R, Garcia Irving M, Varghese Bino A, Grubbs Brendan H, Miki Toshio
Abstract excerpt
Mucopolysaccharidosis type 1 (MPS1) is an inherited lysosomal storage disorder caused by a deficiency in the glycosaminoglycan (GAG)-degrading enzyme α-l-iduronidase (IDUA). In affected patients, the systemic accumulation of GAGs results in skeletal dysplasia, neurological degeneration, multiple organ dysfunction, and early death. Current therapies, including enzyme replacement and bone marrow transplant, improve...
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