Increasing Importance of Stem Cell Gene Therapy in Adenosine Deaminase Deficiency?
Author:
Affiliation:
1. Department of Pediatrics, Division of Allergy/Immunology, Louisiana State University Health Sciences Center, 1542 Tulane Avenue, T8-1, LSUHSC-NO, New Orleans, LA 70112
Publisher
American Society for Microbiology
Subject
Microbiology (medical),Clinical Biochemistry,Immunology,Immunology and Allergy
Link
https://journals.asm.org/doi/pdf/10.1128/CVI.13.3.433-435.2006
Reference35 articles.
1. Aiuti, A. 2004. Gene therapy for adenosine-deaminase-deficient severe combined immunodeficiency. Best Pract. Res. Clin. Haematol.17:505-516.
2. Aiuti, A., S. Slavin, M. Aker, F. Ficara, S. Deola, A. Mortellaro, S. Morecki, G. Andolfi, A. Tabucchi, F. Carlucci, E. Marinello, F. Cattaneo, S. Vai, P. Servida, R. Miniero, M. G. Roncarolo, and C. Bordignon. 2002. Correction of ADA-SCID by stem cell gene therapy combined with nonmyeloablative conditioning. Science296:2410-2413.
3. Blackburn, M. R., M. Aldrich, J. B. Volmer, W. Chen, H. Zhong, S. Kelly, M. S. Hershfield, S. K. Datta, and R. E. Kellems. 2000. The use of enzyme therapy to regulate the metabolic and phenotypic consequences of adenosine deaminase deficiency in mice. J. Biol. Chem.275:32114-32121.
4. Buckley, R. H., S. E. Schiff, R. I. Schiff, L. Markert, L. W. Williams, J. L. Roberts, L. A. Myers, and F. E. Ward. 1999. Hematopoietic stem-cell transplantation for the treatment of severe combined immunodeficiency. N. Engl. J. Med.340:508-516.
5. Cavazzana-Calvo, M., and A. Fischer. 2004. Efficacy of gene therapy for SCID is being confirmed. Lancet364:2155-2156.
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