Liu, David R.,Thompson, David B.,Zuris, John Anthony
申请号：
AU2020233745
公开号：
AU2020233745A1
申请日:
2020.09.18
申请国别(地区):
AU
年份:
2020
代理人:
摘要：
#$%^&*AU2020233745A120201008.pdf#####ABSTRACT Compositions, methods, strategies, kits, and systems for the supercharged proteinmediated 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 functional 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.
