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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Publicado en:Blood
Autores principales: 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
Lenguaje:Inglês
Publicado: American Society of Hematology 2017
Materias:
Gene Therapy
Acceso en línea: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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