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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Salvato in:
Dettagli Bibliografici
Pubblicato in:Blood
Autori principali: Cooper, Aaron R., Lill, Georgia R., Shaw, Kit, Carbonaro-Sarracino, Denise A., Davila, Alejandra, Sokolic, Robert, Candotti, Fabio, Pellegrini, Matteo, Kohn, Donald B.
Natura: Artigo
Lingua:Inglês
Pubblicazione: American Society of Hematology 2017
Soggetti:
Gene Therapy
Accesso online: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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