Establishment of Baboon envelope pseudotyped LV vector system to maintain the stemness in stem cell gene therapy.

2017 Fiscal Year Final Research Report

• Project/Area Number: 15K09676
• Research Category: Grant-in-Aid for Scientific Research (C)
• Allocation Type: Multi-year Fund
• Section: 一般
• Research Field: Pediatrics
• Research Institution: National Center for Child Health and Development
• Principal Investigator: Uchiyama Toru 国立研究開発法人国立成育医療研究センター, 成育遺伝研究部, 室長 (10436107)
• Research Collaborator: Minegishi Tomoko ; Akiba Yumi
• Project Period (FY): 2015-04-01 – 2018-03-31
• Keywords: レンチウイルスベクター / 造血幹細胞遺伝子治療 / 幹細胞性 / ヒヒ内在性レトロウイルス

Outline of Final Research Achievements

An envelope protein of Baboon endogenous retrovirus (BaEV env) can efficiently package the lentivirus (LV) without any cytotoxicity, and we used BaEV env to make the stable packaging cell line for LV vector. LV gag-pol genes encoding structural proteins and rev regulatory gene were transferred into 293T cells to make 293LVgpr cells. Resultant cells expressed gag-pol and rev, and could make the lentivirus particle by the transfection of vector and any kind of envelope plasmids. To make the stable packaging cell line, we then transduce 293LVgpr cells with self-inactivating retroviral vector containing BaEV env. Transduced cells, however, could not produce high titer of LV by the transfection of vector plasmid, because the expression level of BaEV env is not sufficient. We now try to increase the copy number of BaEV env in 293LVgpr cells to increase its expression.

Free Research Field

遺伝子治療