HOME HELP FEEDBACK SUBSCRIPTIONS ARCHIVE SEARCH TABLE OF CONTENTS REGISTER		Author Keyword(s) Vol Page [Advanced]

This Article Full Text Full Text (PDF) Submit a response Alert me when this article is cited Alert me when eLetters are posted Alert me if a correction is posted Citation Map Services Email this link to a friend Similar articles in ADC Online Similar articles in PubMed Add article to my folders Download to citation manager Request Permissions Citing Articles Citing Articles via HighWire Citing Articles via Google Scholar Google Scholar Articles by Steward, C G Articles by Jarisch, A Search for Related Content PubMed PubMed Citation Articles by Steward, C G Articles by Jarisch, A

Haemopoietic stem cell transplantation for genetic disorders

Bone Marrow Transplant Unit, Royal Hospital for Children, Bristol, UK

Correspondence to:
Correspondence to:
Dr C G Steward
Bone Marrow Transplant Unit, Royal Hospital for Children, Upper Maudlin Street, Bristol BS2 8BJ, UK; colin.steward{at}bristol.ac.uk

ABSTRACT
Stem cell transplantation (SCT) is used to cure or greatly ameliorate a wide variety of genetic diseases, ranging from inherent defects of haemopoietic cell production or function to metabolic diseases mostly affecting solid organs. It ranks as one of the most remarkable therapeutic advances of the past 40 years. Despite rapid technological improvements, however, there are still many short term risks and potential long term toxicities. As a consequence, the rapid emergence of alternative therapies (including new drugs, enzyme and gene therapies), necessitate constant re-evaluation of the risk/benefit ratio for each disease and hence the appropriateness of SCT. This review describes the major aspects of the transplant process, indications for transplantation, outcome statistics, and areas where alternative therapies are becoming available.

Abbreviations: GVHD, graft versus host disease; MSD, matched sibling donor; MUD, matched unrelated donor; PCR, polymerase chain reaction; SCID, severe combined immunodeficiency; SCT, stem cell transplantation; TCD, T-cell depletion

Keywords: haematopoietic stem cell transplantation; genetic diseases; inborn; severe combined immunodeficiency; metabolism; inborn errors; chimerism

This article has been cited by other articles:

				J. M.Y. Wong and K. Collins Telomerase RNA level limits telomere maintenance in X-linked dyskeratosis congenita Genes & Dev., October 15, 2006; 20(20): 2848 - 2858. [Abstract] [Full Text] [PDF]