Gene therapy for severe combined immune deficiency

Expert Reviews in Molecular Medicine
Waseem QasimAdrian J Thrasher

Abstract

Infants born with severe combined immune deficiencies are prone to life-threatening infections and, without treatment, do not survive beyond the first year of life. Haematopoietic stem cell transplantation from a fully matched donor offers the possibility of cure. In the absence of a suitable matched donor, haploidentical transplants from a parental donor may be undertaken, but these are associated with more complications and lower success rates. Recently, an alternative therapeutic option based on retroviral gene delivery has been used to correct X-linked severe combined immune deficiency (SCID-X1) and adenosine deaminase deficiency. Clinical trials have established that in situations where ex vivo gene transfer into haematopoietic progenitor cells confers a strong selective advantage, the procedure is a feasible alternative to haploidentical transplantation, with favourable kinetics of immune reconstitution.

Citations

Jan 17, 2016·Annals of the New York Academy of Sciences·Frank J T StaalKarin Pike-Overzet
Apr 27, 2005·Molecular Therapy : the Journal of the American Society of Gene Therapy·Simon N WaddingtonJesus Prieto
Nov 3, 2009·Molecular Human Reproduction·Fuller W BazerKayla Bayless
Feb 24, 2010·Proceedings of the National Academy of Sciences of the United States of America·Steven W ColeTeresa E Seeman
Jun 21, 2013·Science Translational Medicine·Lisa M JohansenGene G Olinger
Oct 21, 2010·Human Reproduction Update·F M MenziesS M Nelson
Mar 13, 2013·Human Reproduction·Simon E ChauRosemary J Keogh
Feb 16, 2011·Human Reproduction·Harmeet SinghGuiying Nie

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