Gene Therapy for Severe Combined Immunodeficiency due to Adenosine Deaminase Deficiency

作者：Montiel Equihua Claudia A; Thrasher Adrian J; Gaspar H Bobby^*

来源：Current Gene Therapy, 2012, 12(1): 57-65.

• Adenosine deaminase
• severe combined immunodeficiency
• gene therapy
• hematopoietic stem cell
• retrovirus
• clinical trial

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.

• 出版日期2012-2