Ronald Cohn,Daria Wojtal,Dwi Kemaladewi,Zhenya Ivakine
申请号：
US15756837
公开号：
US20180243446A1
申请日:
2016.09.01
申请国别(地区):
US
年份:
2018
代理人:
摘要：
In one aspect, the Invention relates to methods and compositions to remove replicate genetic material, such as duplicate copy number variations (CNVs) present in genetic material using targeted endonuclease technology with one guide, such as one single guide RNA. In another aspect, the invention relates to methods and compositions useful for removing replicate or duplicate genetic material. In another aspect the invention is useful for the treatment and prevention of conditions that are caused by replicate genetic material, including duplicate CNVs, such as a certain types of Duchenne Muscular Dystrophy and or MECP2 duplication syndrome. In another aspect the invention provides novel genetically engineered animals with duplicate CNVs useful to screen potential therapies.
