Cytoreductive conditioning intensity predicts clonal diversity in ADA-SCID retroviral gene therapy patients

Retroviral gene therapy has proved efficacious for multiple genetic diseases of the hematopoietic system, but roughly half of clinical gene therapy trial protocols using gammaretroviral vectors have reported leukemias in some of the patients treated. In dramatic contrast, 39 adenosine deaminase–defi...

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Detalhes bibliográficos
Publicado no:Blood
Main Authors: Cooper, Aaron R., Lill, Georgia R., Shaw, Kit, Carbonaro-Sarracino, Denise A., Davila, Alejandra, Sokolic, Robert, Candotti, Fabio, Pellegrini, Matteo, Kohn, Donald B.
Formato: Artigo
Idioma:Inglês
Publicado em: American Society of Hematology 2017
Assuntos:
Gene Therapy
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC5428461/
https://ncbi.nlm.nih.gov/pubmed/28351939
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1182/blood-2016-12-756734
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