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10.1016/j.ymthe.2017.03.018

http://scihub22266oqcxt.onion/10.1016/j.ymthe.2017.03.018
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C5417846!5417846!28366768
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suck abstract from ncbi


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pmid28366768      Mol+Ther 2017 ; 25 (5): 1132-41
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  • Evolving Gene Therapy in Primary Immunodeficiency #MMPMID28366768
  • Thrasher AJ; Williams DA
  • Mol Ther 2017[May]; 25 (5): 1132-41 PMID28366768show ga
  • Prior to the first successful bone marrow transplant in 1968, patients born with severe combined immunodeficiency (SCID) invariably died. Today, with a widening availability of newborn screening, major improvements in the application of allogeneic procedures, and the emergence of successful hematopoietic stem and progenitor cell (HSC/P) gene therapy, the majority of these children can be identified and cured. Here, we trace key steps in the development of clinical gene therapy for SCID and other primary immunodeficiencies (PIDs), and review the prospects for adoption of new targets and technologies.
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