Invention Application
- 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.: US20180371457A1Publication Date: 2018-12-27
- 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 CA 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 CA Cold Spring Harbor
- Main IPC: C12N15/113
- IPC: C12N15/113 ; C12N15/11

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
- US10344282B2 Compositions and methods for modulation of IKBKAP splicing Public/Granted day:2019-07-09
Information query
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