Buckley, Dennis,Winter, Georg,Phillips, Andrew J.,Heffernan, Timothy,Bradner, James,Roberts, Justin,Nabet, Behnam
申请号：
AU2016301196
公开号：
AU2016301196A1
申请日:
2016.08.08
申请国别(地区):
AU
年份:
2018
代理人:
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.
