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Delivery system for functional nucleases
专利权人:
President and Fellows of Harvard College
发明人:
David R. Liu,John Anthony Zuris,David B. Thompson
申请号:
US14462163
公开号:
US09526784B2
申请日:
2014.08.18
申请国别(地区):
US
年份:
2016
代理人:
摘要:
Compositions, methods, strategies, kits, and systems for the supercharged protein-mediated delivery of functional effector proteins into cells in vivo, ex vivo, or in vitro are provided. Compositions, methods, strategies, kits, and systems for delivery of funcational effector proteins using cationic lipids and cationic polymers are also provided. Functional effector proteins include, without limitation, transcriptional modulators (e.g., repressors or activators), recombinases, nucleases (e.g., RNA-programmable nucleases, such as Cas9 proteins; TALE nuclease, and zinc finger nucleases), deaminases, and other gene modifying/editing enzymes. Functional effector proteins include TALE effector proteins, e.g., TALE transcriptional activators and repressors, as well as TALE nucleases. Compositions, methods, strategies, and systems for the delivery of functional effector proteins into cells is useful for therapeutic and research purposes, including, but not limited to, the targeted manipulation of a gene associated with disease, the modulation of the expression level of a gene associated with disease, and the programming of cell fate.
来源网站:
中国工程科技知识中心
来源网址:
http://www.ckcest.cn/home/

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