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pro vyhledávání: '"Melisa Vance"'
Autor:
Aravind Asokan, Telmo Llanga, Giridhar Murlidharan, Brian C. Gilger, Neil C Chungfat, Joanne Kurtzberg, R. Jude Samulski, William D. Bennett, Kenton T. Woodard, Matthew L. Hirsch, Melisa Vance
Publikováno v:
Scientific Reports
Although cord blood transplantation has significantly extended the lifespan of mucopolysaccharidosis type 1 (MPS1) patients, over 95% manifest cornea clouding with about 50% progressing to blindness. As corneal transplants are met with high rejection
Publikováno v:
Molecular Therapy. 24:S40-S41
Mucopolysaccharidosis type 1 (MPS1), also known as Hurler syndrome, is a genetic lysosomal storage disease that results from the loss-of-function mutations present on the L-iduronidase (IDUA) gene. As a consequence, glycosaminoglycans accumulate aber
Autor:
Laurie R. Goodrich, Joanne Kurtzberg, Llanga Telmo, Richard Jude Samulski, Matthew L. Hirsch, Melisa Vance
Publikováno v:
Blood. 126:2041-2041
Mucopolysaccharidosis type 1 (MPS1), also known as Hurler syndrome, is a genetic lysosomal storage disease that results from the loss-of-function mutations present on the L-iduronidase (IDUA) gene. As a consequence, glycosaminoglycans accumulate aber