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

Content Provider	PubMed Central
Author	Candotti, Fabio Shaw, Kit L. Linda, Muul Carbonaro, Denise Robert, Sokolic Choi, Christopher Schurman, Shepherd H. Garabedian, Elizabeth Kesserwan, Chimene Jayashree, Jagadeesh G. Fu, Pei-yu Gschweng, Eric Cooper, Aaron Tisdale, John F. Weinberg, Kenneth I. Crooks, Gay M. Kapoor, Neena Shah, Ami Abdel-azim, Hisham Yu, Xiao-jin Monika, Smogorzewska Wayne, Alan S. Rosenblatt, Howard M. Davis, Carla M. Hanson, Celine Rishi, Radha G. Wang, Xiaoyan David, Gjertson Yang, Otto O. Arumugam, Balamurugan Bauer, Gerhard Ireland, Joanna A. Engel, Barbara C. Podsakoff, Gregory M. Hershfield, Michael S. Blaese, R. Michael Parkman, Robertson Kohn, Donald B.
Copyright Year	2012
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 and remained on ADA enzyme-replacement therapy (ERT) throughout the procedure. Only transient (months), low-level (< 0.01%) gene marking was observed in PBMCs of 2 older subjects (15 and 20 years of age), whereas some gene marking of PBMC has persisted for the past 9 years in 2 younger subjects (4 and 6 years). Six additional subjects were treated using the same gene transfer protocol, but after withdrawal of ERT and administration of low-dose busulfan (65-90 mg/m2). Three of these remain well, off ERT (5, 4, and 3 years postprocedure), with gene marking in PBMC of 1%-10%, and ADA enzyme expression in PBMC near or in the normal range. Two subjects were restarted on ERT because of poor gene marking and immune recovery, and one had a subsequent allogeneic hematopoietic stem cell transplantation. These studies directly demonstrate the importance of providing nonmyeloablative pretransplantation conditioning to achieve therapeutic benefits with gene therapy for ADA-deficient severe combined immunodeficiency.
Related Links	http://dx.doi.org/10.1182/blood-2012-02-400937
Ending Page	3646
Page Count	12
Starting Page	3635
File Format	PDF
ISSN	00064971
e-ISSN	15280020
Journal	Blood
Issue Number	18
Volume Number	120
Language	English
Publisher	American Society of Hematology
Publisher Date	2012-11-01
Access Restriction	Open
Rights Holder	American Society of Hematology
Subject Keyword	Immunology Cell Biology Biochemistry Hematology Research in Higher Education
Content Type	Text
Resource Type	Article
Subject	Cell Biology Hematology Biochemistry Immunology

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