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Gene therapy of severe combined immunodeficiencies
Author(s) -
CavazzanaCalvo Marina,
HaceinBey Salima,
Yates Frank,
de Villartay J. P.,
Le Deist F.,
Fischer A.
Publication year - 2001
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 - 10.1002/1521-2254(200105/06)3:3<201::aid-jgm195>3.0.co;2-z
Subject(s) - genetic enhancement , severe combined immunodeficiency , transgene , haematopoiesis , transplantation , hematopoietic stem cell transplantation , transduction (biophysics) , stem cell , viral vector , immunodeficiency , immunology , biology , disease , medicine , gene , cancer research , immune system , pathology , genetics , biochemistry , recombinant dna
Recent advances in gene transfer in human hematopoietic cells, combined with a better understanding of the genetic aspects of several immunodeficiencies, has offered new opportunities in the domain of gene therapy. Severe combined immunodeficiency (SCID) appear to represent a good model for the application of gene therapy, combining an expected selective advantage for transduced cells, an absence of immunological response to the vector and/or the therapeutic transgene, together with accessibility to hematopoietic stem cells (HSC). Ex vivo retroviral transduction of a therapeutic transgene in HSC prior to transplantation appears to be a particularly effective and long‐lasting means of restoring the expression of a mutated gene in the lymphoid lineage. Furthermore, encouraging therapeutic benefits as a result of a gene therapy protocol for the treatment of X‐linked severe combined immunodeficiencies (SCID‐X1) invites many questions as to the reasons for this therapeutic benefit. This review outlines the results that have been achieved in gene therapy for SCID‐X1, ADA‐SCID as well as other types of SCID, and discusses the possible relationship between the physiopathology of each disease and the success of relevant trials. Copyright © 2001 John Wiley & Sons, Ltd.