Improvements to Cas9-Mediated Mutation
- 技术优势
- High efficiencyTargeted mutagenesisNo off-targets with dCas9 editing
- 技术应用
- Precision gene editing in therapeutic contextsIntroduction of desired traits in cropsGeneration of genetically modified organismsIsogenic cell line generation
- 详细技术说明
- None
- *Abstract
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Cas9 is an RNA-guided DNA endonuclease used to perform targeted genomic manipulations, which can include the error-prone knockout of sequences via non-homologous end joining (NHEJ) and the introduction of precise edits via homology directed repair (HDR). HDR editing shows great promise for a variety of uses, such as generating new cellular immunotherapies, curing genetic disease, and introducing traits into agricultural crops. Yet the efficiency of HDR has lagged behind that of NHEJ, complicating these exciting applications. Additionally, worries have arisen about unintended knockout from off-target NHEJ.
UC Berkeley researchers have found that Cas9 operates by a surprising mechanism, which suggested ways to improve HDR. Taking advantage of this mechanism, researchers found simple methods to dramatically increase the efficiency of HDR, introducing targeted mutations in human cells with frequencies around 60%. Additionally, catalytically inactive Cas9 can be used to make mutations via HDR without attendant error-prone NHEJ. This latter activity allows the precise introduction of mutations with no danger of undesired knockout at off-target sequences.
- *Principal Investigation
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Name: Jacob Ellery Corn
Department:
Name: Christopher Richardson
Department:
- 其他
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Publication
Additional Technologies by these Inventors
Tech ID/UC Case
25181/2016-001-0
Related Cases
2016-001-0
- 国家/地区
- 美国
