Gene Therapy for Severe Combined Immunodeficiency due to Adenosine Deaminase Deficiency

Title:Gene Therapy for Severe Combined Immunodeficiency due to Adenosine Deaminase Deficiency

Volume: 12 Issue: 1

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

Affiliation:

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

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.

Cite this article as:

Claudia A. Montiel-Equihua, Adrian J. Thrasher, H. Bobby Gaspar , Gene Therapy for Severe Combined Immunodeficiency due to Adenosine Deaminase Deficiency, Current Gene Therapy 2012; 12 (1) . https://dx.doi.org/10.2174/156652312799789253