Emerging trends in transplantation of inherited metabolic diseases.

Journal Article (Journal Article;Review)

Allogeneic hematopoietic stem cell transplantation (HSCT) can prolong life and improve its quality in patients with inherited metabolic diseases. HSCT offers a permanent source of enzyme replacement therapy and also might mediate nonhematopoietic cell regeneration or repair. Unrelated cord blood is an exciting newer graft source for treatment of patients with these fatal disorders, providing increased access to donors and significant clinical efficacy, particularly when transplantation is performed in early stages. Pre-transplant performance status is highly predictive of overall survival.

Full Text

Duke Authors

Cited Authors

  • Prasad, VK; Kurtzberg, J

Published Date

  • January 2008

Published In

Volume / Issue

  • 41 / 2

Start / End Page

  • 99 - 108

PubMed ID

  • 18176609

International Standard Serial Number (ISSN)

  • 0268-3369

Digital Object Identifier (DOI)

  • 10.1038/sj.bmt.1705970


  • eng

Conference Location

  • England