Bone marrow transplantation as effective treatment of central nervous system disease in globoid cell leukodystrophy, metachromatic leukodystrophy, adrenoleukodystrophy, mannosidosis, fucosidosis, aspartylglucosaminuria, Hurler, Maroteaux-Lamy, and Sly syndromes, and Gaucher disease type III

William Krivit, Charles Peters, Elsa G. Shapiro

Research output: Contribution to journalReview articlepeer-review

235 Scopus citations

Abstract

Over 400 patients with lysosomal and peroxisomal storage diseases have received hematopoietic stem cell transplantation from normal donors. Without treatment, all of these diseases have an inexorable fate leading to central nervous system deterioration and early death. On the other hand, all of the engrafted hosts have had a remarkable positive clinical improvement in response to normalization of previously deficient enzymatic activity. Survival data for those engrafted indicates continued life-span as long as two decades beyond transplantation. The particular diseases treated in this way are included in this article. The specific indications and methods for transplantation are also included in this article.

Original languageEnglish (US)
Pages (from-to)167-176
Number of pages10
JournalCurrent Opinion in Neurology
Volume12
Issue number2
DOIs
StatePublished - May 17 1999

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