| 研究実績の概要 |
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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