Gene therapy for adenosine deaminase–deficient severe combined immune deficiency: clinical comparison of retroviral vectors and treatment plans

Candotti, Fabio; L. Shaw, Kit; Muul, Linda; A. Carbonaro, Denise; Sokolic, Robert; Choi, Christopher; H. Schurman, Shepherd; Garabedian, Elizabeth; Kesserwan, Chimene; Jayashree Jagadeesh, G.; Fu, Pei-Yu; H. Gschweng, Eric; Cooper, Aaron; F. Tisdale, John; I. Weinberg, Kenneth; M. Crooks, Gay; Kapoor, Neena; J. Shah, Ami; Abdel‐Azim, Hisham; Yu, Xiao-Jin; Smogorzewska, Monika; S. Wayne, Alan; M. Rosenblatt, Howard; M. Davis, Carla; Hanson, Celine; Rishi, Radha; Wang, Xiaoyan; W. Gjertson, David; O. Yang, Otto; Balamurugan, Arumugam; Bauer, Gerhard; A. Ireland, Joanna; C. Engel, Barbara; M. Podsakoff, Gregory; S. Hershfield, Michael; Michael Blaese, R.; Parkman, Robertson; B. Kohn, Donald

doi:10.1182/blood-2012-02-400937

Public

Gene therapy for adenosine deaminase–deficient severe combined immune deficiency: clinical comparison of retroviral vectors and treatment plans

Published in Blood • Sep 13, 2012

Authors:

Fabio Candotti

Kit L. Shaw

Linda Muul

Abstract

We conducted a gene therapy trial in 10 patients with adenosine deaminase (ADA)-deficient severe combined immunodeficiency using 2 slightly different retroviral vectors for the transduction of patients' bone marrow CD34(+) cells. Four subjects were treated without pretransplantation cytoreduction an...

Subject

Adenosine deaminase deficiency

Genetic enhancement

Adenosine deaminase

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