Invention Grant
- Patent Title: Allele-specific therapy for Huntington disease haplotypes
-
Application No.: US16736023Application Date: 2020-01-07
-
Publication No.: US10731160B2Publication Date: 2020-08-04
- Inventor: Amber L. Southwell , Christopher Kay , Michael R. Hayden , Nicholas S. Caron
- Applicant: The University of British Columbia
- Applicant Address: CA Vancouver
- Assignee: The University of British Columbia
- Current Assignee: The University of British Columbia
- Current Assignee Address: CA Vancouver
- Agency: Christensen O'Connor Johnson Kindness PLC
- Main IPC: C12N15/113
- IPC: C12N15/113 ; C12N15/11 ; C07K14/47
Abstract:
The present invention relates to compositions and methods of use thereof for inhibiting mutant HTT mRNA transcription or CAG-expanded HTT protein expression in a cell, comprising contacting the cell with an effective amount of an oligomer targeting a differentiating polymorphism, wherein the differentiating polymorphism is selected from rs72239206, rs363107, rs362313, rs2530595, rs113407847. Specific oligomer sequences are also provided.
Information query
IPC分类:
