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filingDate 2016-10-05-04:00^^<http://www.w3.org/2001/XMLSchema#date>
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publicationDate 2018-07-17-04:00^^<http://www.w3.org/2001/XMLSchema#date>
publicationNumber CN-108291225-A
titleOfInvention Use of single-stranded antisense oligonucleotides in the prevention or treatment of genetic diseases involving trinucleotide repeat expansion
abstract The present invention relates to antisense oligonucleotides (AON) comprising repeating trinucleotide units for the treatment or prevention of hereditary eye diseases, preferably ocular dystrophies caused by RNA toxicity such as Fuchs endothelial corneal dystrophy (FECD) . The oligonucleotides of the invention are used to target expansions of trinucleotide repeat (TNR) sequences present in intronic sequences to prevent disease-associated sequestration of cellular proteins interacting with such TNR expansions.
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