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pro vyhledávání: '"Kevin Murphy R. Parducho"'
Publikováno v:
Transplantation. 103:e182-e187
Background Advances in prenatal screening and early diagnosis of genetic disease will potentially allow for preemptive treatment of anticipated postnatal disease by in utero cell transplantation (IUCT). This strategy carries potential benefits over p
Autor:
Lisa Yanuaria, Kevin Murphy R. Parducho, Brendan H. Grubbs, Natalie S. Rodriguez, Toshio Miki, Irving M. Garcia, Bino Varghese
Publikováno v:
Stem Cells Translational Medicine
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