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Hematopoietic stem cell gene therapy

Part 1. Genetics

1.7. Gene Therapy

Specialist Review

  1. Adrian J. Thrasher1,
  2. Fabio Candotti2

Published Online: 15 NOV 2005

DOI: 10.1002/047001153X.g107206

Encyclopedia of Genetics, Genomics, Proteomics and Bioinformatics

Encyclopedia of Genetics, Genomics, Proteomics and Bioinformatics

How to Cite

Thrasher, A. J. and Candotti, F. 2005. Hematopoietic stem cell gene therapy. Encyclopedia of Genetics, Genomics, Proteomics and Bioinformatics. 1:1.7:92.

Author Information

  1. 1

    Institute of Child Health, London, UK

  2. 2

    National Institutes of Health, Bethesda, MD, USA

Publication History

  1. Published Online: 15 NOV 2005

Abstract

Hematopoiesis is sustained throughout fetal and adult life by hematopoietic stem cells (HSCs) that are defined by their self-renewal capacity, pluripotentiality, and their ability to repopulate myeloablated recipients. HSCs have therefore become important targets for transplantation and somatic gene therapy. Models of HSC gene therapy in mice and large animals have reproducibly demonstrated that a significant proportion of cells participating in long-term engraftment can be stably transduced by integrating vectors based on mammalian retroviruses. Several clinical studies have also shown that conventional gene transfer technology can produce major beneficial therapeutic effects in human patients.

Keywords:

  • gammaretrovirus;
  • lentivirus;
  • hematopoietic stem cell;
  • primary immunodeficiency;
  • severe combined immunodeficiency;
  • adenosine deaminase;
  • chronic granulomatous disease;
  • thalassemia;
  • insertional mutagenesis