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Gene Therapy for Severe Combined Immunodeficiency due to Adenosine Deaminase Deficiency

[ Vol. 12 , Issue. 1 ]

Author(s):

Claudia A. Montiel-Equihua, Adrian J. Thrasher and H. Bobby Gaspar   Pages 57 - 65 ( 9 )

Abstract:


The severe combined immunodeficiency caused by the absence of adenosine deaminase (SCID-ADA) was the first monogenic disorder for which gene therapy was developed. Over 30 patients have been treated worldwide using the current protocols, and most of them have experienced clinical benefit; importantly, in the absence of any vector-related complications. In this document, we review the progress made so far in the development and establishment of gene therapy as an alternative form of treatment for ADA-SCID patients.

Keywords:

Immunodeficiency, Adenosine, Deaminase Deficiency, Adenosine deaminase, severe combined immunodeficiency, gene therapy, hematopoietic stem cell, retrovirus, clinical trial

Affiliation:

Molecular Immunology Unit, UCL Institute of Child Health, 30, Guilford Street, London WC1N 1EH, UK.



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