Cookies on this website

We use cookies to ensure that we give you the best experience on our website. If you click 'Accept all cookies' we'll assume that you are happy to receive all cookies and you won't see this message again. If you click 'Reject all non-essential cookies' only necessary cookies providing core functionality such as security, network management, and accessibility will be enabled. Click 'Find out more' for information on how to change your cookie settings.

During the past 20 years developments in molecular and cellular biology have kindled the hope that one might eventually ameliorate or even cure some serious genetic diseases by repairing or replacing the defective gene. Other articles deal with the formidable problems of isolating pluripotent hematopoietic stem cells; efficiently, safely, and stably transfecting them, and developing transplantation protocols to ensure that the corrected cells supplant the patient's abnormal stem cells after transplantation. Assuming that these hurdles can be overcome, it will also be important to establish the ideal segment of DNA to introduce into stem cells to ensure that, regardless of its position of integration in the genome, the gene in question will be appropriately regulated. In the case of the globin genes this is a particularly difficult task because in order to correct disorders of globin synthesis we need to obtain high levels of stable, tissue- and developmental-stage specific expression. Issues relevant to this problem arising from the analysis of the human beta globin cluster are discussed in the article in this issue by Grosveld. In this article we review our current understanding of how eukaryotic genes might be expressed from their normal chromosomal environment, using the human alpha globin cluster as a specific example. We also discuss how this information might be used in the development of strategies for gene therapy.

Type

Journal article

Journal

Seminars in hematology

Publication Date

04/1998

Volume

35

Pages

93 - 104

Addresses

Institute of Molecular Medicine, John Radcliffe Hospital, Oxford, United Kingdom.

Keywords

Animals, Mice, Transgenic, Humans, Mice, alpha-Thalassemia, Disease Models, Animal, Globins, Gene Therapy, Chromosome Mapping, Gene Expression, Mutation, Multigene Family