Invention Grant
- Patent Title: Use of single-stranded antisense oligonucleotide in prevention or treatment of genetic diseases involving a trinucleotide repeat expansion
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Application No.: US15765864Application Date: 2016-10-05
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Publication No.: US10760076B2Publication Date: 2020-09-01
- Inventor: Peter Adamson , Janne Juha Turunen , Gerardus Johannes Platenburg
- Applicant: ProQR Therapeutics II B.V.
- Applicant Address: NL Leiden
- Assignee: ProQR Therapeutics II B.V.
- Current Assignee: ProQR Therapeutics II B.V.
- Current Assignee Address: NL Leiden
- Agency: Fish & Richardson P.C.
- Priority: com.zzzhc.datahub.patent.etl.us.BibliographicData$PriorityClaim@78b267ff com.zzzhc.datahub.patent.etl.us.BibliographicData$PriorityClaim@7e459c02
- International Application: PCT/EP2016/073817 WO 20161005
- International Announcement: WO2017/060317 WO 20170413
- Main IPC: C12N15/113
- IPC: C12N15/113 ; A61P27/02 ; A61K9/00 ; A61K31/7105
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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