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Globin gene transfer for the treatment of severe hemoglobinopathies: a paradigm for stem cell‐based gene therapy
Author(s) -
Sadelain Michel
Publication year - 2002
Publication title -
the journal of gene medicine
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 0.689
H-Index - 91
eISSN - 1521-2254
pISSN - 1099-498X
DOI - 10.1002/jgm.266
Subject(s) - genetic enhancement , stem cell , transgene , homing (biology) , haematopoiesis , biology , gene , hematopoietic stem cell , globin , computational biology , stem cell therapy , severe combined immunodeficiency , repopulation , genetics , ecology
The prospect of treating blood disorders with genetically modified stem cells is highly promising. This therapeutic approach, however, raises a number of fundamental biological questions, spanning several research fields. Further investigation is required to better understand how to isolate and efficiently transduce hematopoietic stem cells (HSCs), while preserving optimal homing and self‐renewing properties; how to design safe vectors permitting controlled expression of the transgene products; and how to promote host repopulation by engrafted HSCs. This article addresses basic issues in stem cell‐based gene therapy from the perspective of regulating transgene expression, taking globin gene transfer for the treatment of severe hemoglobinopathies as a paradigm. Copyright © 2002 John Wiley & Sons, Ltd.