发明名称 OLIGONUCLEOTIDES FOR GENOMIC DNA EDITING
摘要 A method for making a change in an endogenous chromosomal DNA sequence of a mammalian cell, comprising steps of: (i) introducing into said cell an oligonucleotide having a sequence that is complementary to the chromosomal DNA sequence and that includes the change; (ii) allowing sufficient time for the cell to incorporate the change into the endogenous chromosomal DNA sequence through endogenous nucleic acid modifying pathways; and (iii) identifying the presence of the change in the chromosomal DNA sequence. The invention is particularly useful for correcting mutations in the CFTR gene.
申请公布号 WO2016062886(A1) 申请公布日期 2016.04.28
申请号 WO2015EP74675 申请日期 2015.10.23
申请人 PROQR THERAPEUTICS II B.V. 发明人 HOLKERS, MAARTEN;SWILDENS, JIM
分类号 C12N15/11;A61K31/7088 主分类号 C12N15/11
代理机构 代理人
主权项
地址