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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Dades bibliogràfiques
Publicat a:Blood
Autors principals: Cooper, Aaron R., Lill, Georgia R., Shaw, Kit, Carbonaro-Sarracino, Denise A., Davila, Alejandra, Sokolic, Robert, Candotti, Fabio, Pellegrini, Matteo, Kohn, Donald B.
Format: Artigo
Idioma:Inglês
Publicat: American Society of Hematology 2017
Matèries:
Gene Therapy
Accés en línia: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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