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

• Автори: Naoya Uchida (Автор), Molly E Evans (Автор), Matthew M Hsieh (Автор), Aylin C Bonifacino (Автор), Allen E Krouse (Автор), Mark E Metzger (Автор), Stephanie E Sellers (Автор), Cynthia E Dunbar (Автор), Robert E Donahue (Автор), John F Tisdale (Автор)
• Формат: Книга
• Опубліковано: Elsevier, 2013-01-01T00:00:00Z.
• Предмети: article
• Онлайн доступ: 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...