USE OF SINGLE-STRANDED ANTISENSE OLIGONUCLEOTIDE IN PREVENTION OR TREATMENT OF GENETIC DISEASES INVOLVING A TRINUCLEOTIDE REPEAT EXPANSION

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专利权人:

ProQR Therapeutics II B.V.

发明人:

Adamson Peter,Turunen Janne Juha,Platenburg Gerardus Johannes

申请号：

US201615765864

公开号：

US2018282724(A1)

申请日:

2016.10.05

申请国别(地区):

美国

年份:

2018

代理人:

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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中国工程科技知识中心

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