发明名称 |
TUNABLE ENDOGENOUS PROTEIN DEGRADATION |
摘要 |
The present invention provides a means to modulate gene expression in vivo in a manner that avoids problems associated with CRISPR endogenous protein knock-out or knock-in strategies and strategies that provide for correction, or alteration, of single nucleotides. The invention includes inserting into the genome a nucleotide encoding a heterobifunctional compound targeting protein (dTAG) in-frame with the nucleotide sequence of a gene encoding an endogenously expressed protein of interest which, upon expression, produces an endogenous protein-dTAG hybrid protein. This allows for targeted protein degradation of the dTAG and the fused endogenous protein using a heterobifunctional compound. |
申请公布号 |
WO2017024319(A1) |
申请公布日期 |
2017.02.09 |
申请号 |
WO2016US46089 |
申请日期 |
2016.08.08 |
申请人 |
DANA-FARBER CANCER INSTITUTE, INC. |
发明人 |
BUCKLEY, Dennis;WINTER, Georg;PHILLIPS, Andrew J.;HEFFERNAN, Timothy;BRADNER, James;ROBERTS, Justin;NABET, Behnam |
分类号 |
C07K16/00;A61K35/17;A61P35/00 |
主分类号 |
C07K16/00 |
代理机构 |
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代理人 |
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主权项 |
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地址 |
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