期刊
CURRENT OPINION IN IMMUNOLOGY
卷 24, 期 5, 页码 580-584出版社
CURRENT BIOLOGY LTD
DOI: 10.1016/j.coi.2012.08.008
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资金
- European Commission: Advanced Cell-based Therapies for the treatment of Primary ImmunoDeficiency (Cell-PID) [HEALTH F5-2010-261387]
- Italian Telethon Foundation
- European Research Council (ERC): Stem cell and gene therapy approaches for inherited diseases with unsatisfying or no therapeutic option [ERC-2010-AdG_20100317]
Over 60 patients affected by SCID due to IL2RG deficiency (SCID-X1) or adenosine deaminase (ADA)-SCID have received hematopoietic stem cell gene therapy in the past 15 years using gammaretroviral vectors, resulting in immune reconstitution and clinical benefit in the majority of them. However, the occurrence of insertional oncogenesis in the SCID-X1 trials has led to the development of new clinical trials based on integrating vectors with improved safety design as well as investigation on new technologies for highly efficient gene targeting and site-specific gene editing. Here we will present the experience and perspectives of gene therapy for SCID-X1 and ADA-SCID and discuss the pros and cons of gene therapy in comparison to allogeneic transplantation.
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