发明名称 |
RNA-Guided Human Genome Engineering |
摘要 |
A method of altering a eukaryotic cell is provided including transfecting the eukaryotic cell with a nucleic acid encoding RNA complementary to genomic DNA of the eukaryotic cell, transfecting the eukaryotic cell with a nucleic acid encoding an enzyme that interacts with the RNA and cleaves the genomic DNA in a site specific manner, wherein the cell expresses the RNA and the enzyme, the RNA binds to complementary genomic DNA and the enzyme cleaves the genomic DNA in a site specific manner. |
申请公布号 |
US2014342457(A1) |
申请公布日期 |
2014.11.20 |
申请号 |
US201414319171 |
申请日期 |
2014.06.30 |
申请人 |
President and Fellows of Harvard College |
发明人 |
MALI Prashant G.;CHURCH George M.;Yang Luhan |
分类号 |
C12N15/01;C12N15/82;C12N15/85;C12N15/81 |
主分类号 |
C12N15/01 |
代理机构 |
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代理人 |
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主权项 |
1. A method of modulating expression of a target nucleic acid in a cell comprising
providing to the cell a guide RNA complementary to the target nucleic acid sequence, providing to the cell a Cas9 protein having inactive nuclease domains that interacts with the guide RNA and binds to the target nucleic acid sequence in a site specific manner, wherein the Cas 9 protein having inactive nuclease domains includes a transcriptional activator or repressor domain as a fusion protein for modulating target nucleic acid expression in vivo, wherein the guide RNA and the Cas9 protein including the transcriptional activator or repressor domain co-localize to the target nucleic acid sequence and wherein the transcriptional activator or repressor domain modulates expression of the target nucleic acid. |
地址 |
Cambridge MA US |