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

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 t...

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Bibliographic Details
Main Authors: Naoya Uchida, Molly E Evans, Matthew M Hsieh, Aylin C Bonifacino, Allen E Krouse, Mark E Metzger, Stephanie E Sellers, Cynthia E Dunbar, Robert E Donahue, John F Tisdale
Format: Article
Language:English
Published: Elsevier 2013-01-01
Series:Molecular Therapy: Nucleic Acids
Subjects:
CD34+ cells
hematopoietic stem cell transplantation
large animal model
lentiviral vector
Online Access:http://www.sciencedirect.com/science/article/pii/S2162253116301810

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http://www.sciencedirect.com/science/article/pii/S2162253116301810

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