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Integration-specific In Vitro Evaluation of Lentivirally Transduced Rhesus CD34(+) Cells Correlates With In Vivo Vector Copy Number

Hematopoietic stem cell (HSC) gene therapy using integrating vectors has a potential leukemogenic risk due to insertional mutagenesis. To reduce this risk, a limitation of ≤2 average vector copy number (VCN) per cell is generally accepted. We developed an assay for VCN among transduced CD34(+) cells...

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Main Authors: Uchida, Naoya, Evans, Molly E, Hsieh, Matthew M, Bonifacino, Aylin C, Krouse, Allen E, Metzger, Mark E, Sellers, Stephanie E, Dunbar, Cynthia E, Donahue, Robert E, Tisdale, John F
Format: Artigo
Jezik:Inglês
Izdano: Nature Publishing Group 2013
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Online dostop:https://ncbi.nlm.nih.gov/pmc/articles/PMC4098567/
https://ncbi.nlm.nih.gov/pubmed/24045711
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mtna.2013.49
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