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filingDate 2016-10-05-04:00^^<http://www.w3.org/2001/XMLSchema#date>
grantDate 2020-09-01-04:00^^<http://www.w3.org/2001/XMLSchema#date>
inventor http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_8ccb5eb5709377a63e1122373d9e6511
http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_7a7fe8fce720fd2d1c76f2f0beebadeb
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publicationDate 2020-09-01-04:00^^<http://www.w3.org/2001/XMLSchema#date>
publicationNumber US-10760076-B2
titleOfInvention Use of single-stranded antisense oligonucleotide in prevention or treatment of genetic diseases involving a trinucleotide repeat expansion
abstract 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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