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公开(公告)号：EP3359667A1
公开(公告)日：2018-08-15
申请号：EP16779049.2
申请日：2016-10-05
IPC分类号： C12N15/113
CPC分类号： C12N15/113 , A61K9/0019 , A61K9/0048 , A61K31/7105 , A61P27/02 , C12N2310/11 , C12N2310/315 , C12N2310/321 , C12N2320/32 , C12N2310/3521
摘要： The invention relates to antisense oligonucleotides (AONs) comprising repetitive trinucleotide units for use in the treatment or prevention of genetic eye diseases, preferably eye dystrophy disorders caused by RNA toxicity such as Fuch's Endothelial Corneal Dystrophy (FECD). The oligonucleotides of the present invention are used to target trinucleotide repeat (TNR) sequence expansions present in intron sequences, to prevent the disease-related sequestration of cellular proteins that interact with such TNR expansions.
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公开(公告)号：EP4301856A2
公开(公告)日：2024-01-10
申请号：EP22710098.9
申请日：2022-03-04
发明人： VAN MIERLO, Elisabeth Laurentina Wilhelmina Maria , PLATENBURG, Gerardus Johannes , YILMAZ-ELIS, Aliye Seda
IPC分类号： C12N15/113 , A61K31/713
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公开(公告)号：EP3712269A1
公开(公告)日：2020-09-23
申请号：EP20169140.9
申请日：2015-12-17
IPC分类号： C12N15/113
摘要： RNA editing is achieved using oligonucleotide constructs comprising a sequence that is sufficiently complementary to the target RNA sequence to bind by nucleobase pairing to said target RNA, and recognized by an RNA editing entity that is naturally present in said eukaryotic cell. The RNA editing entity, such as ADAR, is redirected to a preselected target site, thereby promoting editing of preselected nucleotide residues in a region of the target RNA.
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公开(公告)号：EP4003291A1
公开(公告)日：2022-06-01
申请号：EP20743148.7
申请日：2020-07-24
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公开(公告)号：EP3947678A1
公开(公告)日：2022-02-09
申请号：EP20719120.6
申请日：2020-03-27
IPC分类号： C12N15/113 , A61K31/7125 , A61K31/712 , C07H21/02 , A61P31/00
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