[Bone marrow transplantation for lysosomal storage diseases].
Since a successful bone marrow transplantation (BMT) for Hurler's disease reported in 1981, BMT has been established as an effective treatment for lysosomal storage diseases such as mucopolysaccharidosis or lipidosis. In general, BMT can improve the somatic disease, but the neurologic and intelligence outcomes vary widely. The understanding on whether transfer of a lysosomal hydrolase to CNS neurons can occur is indispensable to a proper prediction on CNS improvement. Furthermore the long-term therapeutic effects of BMT can be subject to multiple factors including a level of biochemical improvement, a reversibility of affected tissues, or a degree of advanced disease prior to BMT. Thus, an accumulation of clinical and biochemical follow-up study is needed to understand the therapeutic efficacy and consequence of BMT for lysosomal storage diseases.
Imaizumi
M
M
Department of Pediatrics, Tohoku University School of Medicine.
D016026:Bone Marrow Transplantation / Q000379:methods; D006801:Humans; D016464:Lysosomal Storage Diseases / Q000628:therapy*