Invention Grant
- Patent Title: RNAi induced huntingtin gene suppression
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Application No.: US16193908Application Date: 2018-11-16
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Publication No.: US10767180B2Publication Date: 2020-09-08
- Inventor: Pavlina Stefanova Konstantinova , Jana Miniariková
- Applicant: uniQure IP B.V.
- Applicant Address: NL Amsterdam
- Assignee: UNIQURE IP B.V.
- Current Assignee: UNIQURE IP B.V.
- Current Assignee Address: NL Amsterdam
- Agency: Foley & Lardner LLP
- Agent Sunit Talapatra
- Priority: com.zzzhc.datahub.patent.etl.us.BibliographicData$PriorityClaim@2f1cba
- Main IPC: C12N15/113
- IPC: C12N15/113 ; A61K31/713

Abstract:
The present invention provides for a double stranded RNA comprising a first RNA sequence and a second RNA sequence wherein the first and second RNA sequence are substantially complementary, wherein the first RNA sequence has a sequence length of at least 19 nucleotides and is substantially complementary to SEQ ID NO. 1. Said double stranded RNA is for use in inducing RNAi against Huntingtin exon 1 sequences. The double stranded RNA of to the invention was capable of reducing neuronal cell death and huntingtin aggregates in an animal model.
Public/Granted literature
- US20190144860A1 RNAI INDUCED HUNTINGTIN GENE SUPPRESSION Public/Granted day:2019-05-16
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