Bone marrow transplantation in lysosomal storage diseases: a prelude to gene-insertion therapy.
Allogeneic bone marrow transplantation is a therapeutic option for enzyme replacement in children with lysosomal storage diseases, for which no current conventional treatments are curative. Although marrow allografting is costly, intensive, and not risk free, strides are being made in the management and prevention of procedure-related complications.
Yeager
A M
AM
Johns Hopkins Oncology Center, Baltimore, MD 21205.
D016026:Bone Marrow Transplantation / Q000639:trends*; D002648:Child; D015316:Genetic Therapy; D005087:Glucan 1,4-alpha-Glucosidase / Q000378:metabolism*; D006008:Glycogen Storage Disease / Q000628:therapy*; D006801:Humans
D005087:Glucan 1,4-alpha-Glucosidase