Guschin, Dmitry,Holmes, Michael C.,Paschon, David,Tam, Philip
申请号：
AU2012286901
公开号：
AU2012286901A1
申请日:
2012.07.25
申请国别(地区):
AU
年份:
2014
代理人:
摘要：
Nucleases and methods of using these nucleases for alteration of a CFTR gene and generation of cells and animal models. In one aspect, described herein is a zinc-finger protein (ZFP) that binds to target site in a CFTR gene in a genome, wherein the ZFP comprises one or more engineered zinc-finger binding domains. In one embodiment, the ZFP is a zinc-finger nuclease (ZFN) that cleaves a target genomic region of interest, wherein the ZFN comprises one or more engineered zinc-finger binding domains and a nuclease cleavage domain or cleavage half-domain. Cleavage domains and cleavage half domains can be obtained, for example, from various restriction endonucleases and/or homing endonucleases. In one embodiment, the cleavage half-domains are derived from a Type IIS restriction endonuclease (e.g., Fok I). In certain embodiments, the zinc finger domain recognizes a target site in a CFTR gene.
