公开(公告)号：EP3262170A1

公开(公告)日：2018-01-03

申请号：EP16707414.5

申请日：2016-02-26

申请人： ProQR Therapeutics II B.V.

发明人： BIASUTTO, Patricia Coromoto ,  CHAN, Hee, Lam

IPC分类号： C12N15/113 ,  A61K31/712 ,  A61K31/7125

CPC分类号： C12N15/113 ,  A61K31/7125 ,  C12N2310/11 ,  C12N2310/315 ,  C12N2310/346 ,  C12N2320/33 ,  C12N2330/51 ,  C12N2310/321 ,  C12N2310/3521

摘要： Antisense oligonucleotides target the mutation in intron 26 of the CEP290 gene and reduce inclusion of the aberrant exon into the CEP290 mRNA. The oligonucleotides include no more than 3 consecutive guanosines, have no more than 60% guanosine nucleobases, include at most one CpG sequence, and/or do not have the potential to form a hairpin comprising 3 or more consecutive complementary base pairs.