Generation of episomal RNA viral vector for stem cell gene therapy

2017 Fiscal Year Final Research Report

• Project/Area Number: 16K19069
• Research Category: Grant-in-Aid for Young Scientists (B)
• Allocation Type: Multi-year Fund
• Research Field: Human genetics
• Research Institution: Kyoto University
• Principal Investigator: Makino Akiko 京都大学, ウイルス・再生医科学研究所, 助教 (30571145)
• Project Period (FY): 2016-04-01 – 2018-03-31
• Keywords: ウイルスベクター / 幹細胞

Outline of Final Research Achievements

Borna disease virus (BoDV) is an RNA virus persistently infecting stem cells. In this study, we aimed to construct a long-term stable gene transfer system that realizes stem cell therapy using the virus. The BoDV vector expressing IL2RG, which is the causative gene of X-SCID, was prepared and inoculated into hematopoietic stem cells. This vector stably expressed the target gene, however, the transduction efficiency was low. The vector in which the G protein was replaced with CnBV-1 increased the efficiency by 15-fold as compared with BoDV. The improved vector significantly increased the efficiency of transduction into stem cells including iPS cells. BoDV vector with X/P gene of VSBV also increased the transduction efficiency. The improved BoDV vector prepared in this study could be a new platform for stem cell gene therapy.

Free Research Field

ウイルス学