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Current Gene Therapy

Editor-in-Chief

ISSN (Print): 1566-5232
ISSN (Online): 1875-5631

Gene Therapy for Severe Combined Immunodeficiency due to Adenosine Deaminase Deficiency

Author(s): Claudia A. Montiel-Equihua, Adrian J. Thrasher, H. Bobby Gaspar

Volume 12 , Issue 1 , 2012

Page: [57 - 65] Pages: 9

DOI: 10.2174/156652312799789253

Price: $65

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


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