Preclinical development and qualification of ZFN-mediated CCR5 disruption in human hematopoietic stem/progenitor cells

Gene therapy for HIV-1 infection is a promising alternative to lifelong combination antiviral drug treatment. Chemokine receptor 5 (CCR5) is the coreceptor required for R5-tropic HIV-1 infection of human cells. Deletion of CCR5 renders cells resistant to R5-tropic HIV-1 infection, and the potential...

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Pubblicato in:Mol Ther Methods Clin Dev
Autori principali: DiGiusto, David L, Cannon, Paula M, Holmes, Michael C, Li, Lijing, Rao, Anitha, Wang, Jianbin, Lee, Gary, Gregory, Philip D., Kim, Kenneth A, Hayward, Samuel B, Meyer, Kathleen, Exline, Colin, Lopez, Evan, Henley, Jill, Gonzalez, Nancy, Bedell, Victoria, Stan, Rodica, Zaia, John A
Natura: Artigo
Lingua:Inglês
Pubblicazione: Nature Publishing Group 2016
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Article
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC5102145/
https://ncbi.nlm.nih.gov/pubmed/27900346
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mtm.2016.67
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