Invention Grant
- Patent Title: Oligonucleotides for treating expanded repeat diseases
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Application No.: US14360890Application Date: 2012-11-30
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Publication No.: US10066228B2Publication Date: 2018-09-04
- Inventor: Peter Linsley , Brian James Leppert , Gunnar J. Hanson
- Applicant: Sarepta Therapeutics, Inc.
- Applicant Address: US MA Cambridge
- Assignee: SAREPTA THERAPEUTICS, INC.
- Current Assignee: SAREPTA THERAPEUTICS, INC.
- Current Assignee Address: US MA Cambridge
- Agency: Lathrop Gage LLP
- Agent Brian C. Trinque
- International Application: PCT/US2012/067470 WO 20121130
- International Announcement: WO2013/082548 WO 20130606
- Main IPC: C12N15/113
- IPC: C12N15/113 ; C07H21/04 ; C12Q1/6883 ; C12N15/11 ; C07F9/6558 ; C07F9/6561

Abstract:
The invention provides for a method for selectively reducing the expression of a mutant mRNA and/or protein having an expanded nucleotide repeat relative to a wild-type mRNA, comprising contacting a cell with an antisense oligonucleotide of sufficient length and complementarity to the expanded nucleotide repeat. More particularly it relates to selectively reducing the expression of mutant Huntington protein associated with Huntington's disease. The antisense oligonucleotide comprising either a nucleotide or a repeated three nucleotide sequence as defined in the claims.
Public/Granted literature
- US20140303238A1 OLIGONUCLEOTIDES FOR TREATING EXPANDED REPEAT DISEASES Public/Granted day:2014-10-09
Information query
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