Peter Adamson,Janne Juha Turunen,Gerardus Johannes Platenburg
申请号：
US15765864
公开号：
US20180282724A1
申请日:
2016.10.05
申请国别(地区):
US
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.
