Gene Therapy for Severe Combined Immunodeficiency due to Adenosine Deaminase Deficiency
Claudia A. Montiel-Equihua, Adrian J. Thrasher, H. Bobby Gaspar.
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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