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    Increased longevity and metabolic correction following syngeneic BMT in a murine model of mucopolysaccharidosis type I

    Mucopolysaccharidosis type I (MPS I) is an autosomal recessive inherited disease caused by deficiency of the glycosidase α-L-iduronidase (IDUA). Deficiency of IDUA leads to lysosomal accumulation of glycosaminogl...

    D A Wolf, A W Lenander, Z Nan, E A Braunlin… in Bone Marrow Transplantation (2012)