Invention Grant
- Patent Title: Compositions and methods for modulation of IKBKAP splicing
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Application No.: US15993090Application Date: 2018-05-30
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Publication No.: US10344282B2Publication Date: 2019-07-09
- Inventor: C. Frank Bennett , Frank Rigo , Adrian R. Krainer , Rahul Sinha
- Applicant: Ionis Pharmaceuticals, Inc. , Cold Spring Harbor Laboratory
- Applicant Address: US CA Carlsbad US NY Cold Spring Harbor
- Assignee: IONIS PHARMACEUTICALS, INC.,COLD SPRING HARBOR LABORATORY
- Current Assignee: IONIS PHARMACEUTICALS, INC.,COLD SPRING HARBOR LABORATORY
- Current Assignee Address: US CA Carlsbad US NY Cold Spring Harbor
- Agency: Michael Best & Friedrich LLP
- Main IPC: C12N15/11
- IPC: C12N15/11 ; C12N15/113

Abstract:
The present disclosure provides compounds comprising oligonucleotides complementary to a portion of the IKBKAP gene. Certain such compounds are useful for hybridizing to a portion of the IKBKAP gene, including but not limited to a portion of the IKBKAP gene in a cell. In certain embodiments, such hybridization results in modulation of splicing of the IKBKAP gene. In certain embodiments, the IKBKAP gene includes a mutation that results in defective splicing and a truncated IKAP protein. In certain embodiments, hybridization of oligonucleotides complementary to a portion of the IKBKAP gene results in a decrease in the amount of defective splicing and truncated IKAP protein. In certain embodiments, hybridization of oligonucleotides complementary to a portion of the IKBKAP gene results in an increase in the amount of normal splicing and functional, full-length IKAP protein. In certain embodiments, oligonucleotides are used to treat Familial Dysautonomia.
Public/Granted literature
- US20180371457A1 COMPOSITIONS AND METHODS FOR MODULATION OF IKBKAP SPLICING Public/Granted day:2018-12-27
Information query
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