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

• Main Authors: Naoya Uchida (Author), Molly E Evans (Author), Matthew M Hsieh (Author), Aylin C Bonifacino (Author), Allen E Krouse (Author), Mark E Metzger (Author), Stephanie E Sellers (Author), Cynthia E Dunbar (Author), Robert E Donahue (Author), John F Tisdale (Author)
• Format: Book
• Published: Elsevier, 2013-01-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

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...