Gaucher's disease. Enzyme therapy is not enough.
Grabowski
G A
GA
Children's Hospital Research Foundation, Cincinnati, Ohio 45229, USA. greg.grabowski@chmcc.org
D000818:Animals; D016026:Bone Marrow Transplantation; D005776:Gaucher Disease / Q000188:drug therapy / Q000201:enzymology / Q000628:therapy*; D015316:Genetic Therapy; D005962:Glucosylceramidase / Q000378:metabolism / Q000627:therapeutic use*; D006801:Humans; D051379:Mice; D018345:Mice, Knockout
D005962:Glucosylceramidase