2018 Fiscal Year Annual Research Report
CRISPR Cas9 protein delivery via virus-like particles for in vivo treatment of Duchenne muscular dystrophy
| Project/Area Number |
17K15048
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| Research Institution | Kyoto University |
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Principal Investigator |
Gee PeterDavid 京都大学, iPS細胞研究所, 特定研究員 (00754227)
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| Project Period (FY) |
2017-04-01 – 2019-03-31
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| Keywords | CRISPR / Genome editing / Delivery / Virus-like particles / DMD |
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| Outline of Annual Research Achievements |
CRISPR-Cas9 is a powerful genome editing tool that holds tremendous potential for treating genetic diseases in humans, such as Duchenne muscular dystrophy (DMD). DMD is a muscle wasting disease that results from mutations in the dystrophin gene. We have developed a dimerization based packaging system to incorporate Cas9 protein into virus-like particles (VLPs) mediated through a chemical ligand induced interaction with a viral structural protein. The resulting RNP-containing VLPs were delivered onto DMD patient iPS cells and reached greater than 40% indel induction. In sum, our VLP-based RNP delivery system may serve as a useful tool for both in vitro and in vivo genome editing in the future.
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Research Products
(2 results)
