Gene therapy for severe combined immune deficiency.

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.

journal_name

Expert Rev Mol Med

authors

Qasim W,Gaspar HB,Thrasher AJ

doi

10.1017/S1462399404007884

keywords:

subject

Has Abstract

pub_date

2004-07-02 00:00:00

pages

1-15

issue

13

issn

1462-3994

pii

S1462399404007884

journal_volume

6

pub_type

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