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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Bibliografische gegevens
Gepubliceerd in:Blood
Hoofdauteurs: Cooper, Aaron R., Lill, Georgia R., Shaw, Kit, Carbonaro-Sarracino, Denise A., Davila, Alejandra, Sokolic, Robert, Candotti, Fabio, Pellegrini, Matteo, Kohn, Donald B.
Formaat: Artigo
Taal:Inglês
Gepubliceerd in: American Society of Hematology 2017
Onderwerpen:
Gene Therapy
Online toegang: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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