Skip to main content

Measurements from normal umbilical cord blood of four lysosomal enzymatic activities: alpha-L-iduronidase (Hurler), galactocerebrosidase (globoid cell leukodystrophy), arylsulfatase A (metachromatic leukodystrophy), arylsulfatase B (Maroteaux-Lamy).

Publication ,  Journal Article
deGasperi, R; Raghavan, SS; Sosa, MG; Kolodny, EH; Carrier, C; Rubenstein, P; Peters, C; Wagner, J; Kurtzberg, J; Krivit, W
Published in: Bone Marrow Transplant
March 2000

Umbilical cord blood (UCB) has received increasing attention as a source of unrelated hematopoietic stem cells for transplantation. Lysosomal diseases have been effectively treated and normal enzymatic activity has occurred subsequent to engraftment using UCB. The use of donor cells with normal amounts of enzyme, rather than those from carriers whose level may be 50% or less, is an obvious goal. The frequency of such heterozygotes varies from 1:10 to 1:140 or lower depending upon the disease at issue. We assayed the levels of lysosomal enzymes in normal UCB in random samples as well as those used for transplantation. We measured the following enzymatic activities: alpha-l-iduronidase (Hurler), galactocerebrosidase (globoid cell leuko- dystrophy) and arylsulfatase A (metachromatic leukodystrophy). For the latter, levels of activity in UCB are comparable to those found in adult blood. In the case of arylsulfatase B (Maroteaux-Lamy) a level lower than adult level was found. An informed choice by the transplanting physician based on the activity of the relevant enzyme in the UCB donor will provide a better opportunity for an improved prognosis for more complete correction of the recipient's primary disease. Bone Marrow Transplantation (2000) 25, 541-544.

Duke Scholars

Published In

Bone Marrow Transplant

DOI

ISSN

0268-3369

Publication Date

March 2000

Volume

25

Issue

5

Start / End Page

541 / 544

Location

England

Related Subject Headings

  • N-Acetylgalactosamine-4-Sulfatase
  • Lysosomes
  • Leukocytes
  • Kinetics
  • Infant, Newborn
  • Immunology
  • Iduronidase
  • Humans
  • Galactosylceramidase
  • Fetal Blood
 

Citation

APA
Chicago
ICMJE
MLA
NLM
deGasperi, R., Raghavan, S. S., Sosa, M. G., Kolodny, E. H., Carrier, C., Rubenstein, P., … Krivit, W. (2000). Measurements from normal umbilical cord blood of four lysosomal enzymatic activities: alpha-L-iduronidase (Hurler), galactocerebrosidase (globoid cell leukodystrophy), arylsulfatase A (metachromatic leukodystrophy), arylsulfatase B (Maroteaux-Lamy). Bone Marrow Transplant, 25(5), 541–544. https://doi.org/10.1038/sj.bmt.1702185
deGasperi, R., S. S. Raghavan, M. G. Sosa, E. H. Kolodny, C. Carrier, P. Rubenstein, C. Peters, J. Wagner, J. Kurtzberg, and W. Krivit. “Measurements from normal umbilical cord blood of four lysosomal enzymatic activities: alpha-L-iduronidase (Hurler), galactocerebrosidase (globoid cell leukodystrophy), arylsulfatase A (metachromatic leukodystrophy), arylsulfatase B (Maroteaux-Lamy).Bone Marrow Transplant 25, no. 5 (March 2000): 541–44. https://doi.org/10.1038/sj.bmt.1702185.

Published In

Bone Marrow Transplant

DOI

ISSN

0268-3369

Publication Date

March 2000

Volume

25

Issue

5

Start / End Page

541 / 544

Location

England

Related Subject Headings

  • N-Acetylgalactosamine-4-Sulfatase
  • Lysosomes
  • Leukocytes
  • Kinetics
  • Infant, Newborn
  • Immunology
  • Iduronidase
  • Humans
  • Galactosylceramidase
  • Fetal Blood