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 |
|