Invention Grant
- Patent Title: Methods and compositions for reducing pathogenic isoforms
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Application No.: US17932996Application Date: 2022-09-16
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Publication No.: US12043832B2Publication Date: 2024-07-23
- Inventor: Andrew Findlay , Conrad Weihl
- Applicant: Washington University
- Applicant Address: US MO St. Louis
- Assignee: Washington University
- Current Assignee: Washington University
- Current Assignee Address: US MO St. Louis
- Agency: Polsinelli PC
- Main IPC: C12N15/113
- IPC: C12N15/113 ; A61P21/00

Abstract:
Among the various aspects of the present disclosure is the provision of compositions and methods for selectively reducing pathogenic isoforms (e.g., DNAJB6) in a subject having a neuromuscular disorder. An aspect of the present disclosure provides for selectively reducing DNAJB6 in a subject having a neuromuscular disorder (e.g., limb-girdle muscular dystrophy D1 (LGMD-D1)) comprising administering an amount of a DNAJB6-targeting antisense oligonucleotide (ASO) sufficient to reduce the expression of DNAJB6 compared to the subject prior to being administered the DNAJB6-targeting ASO.
Public/Granted literature
- US20230079754A1 METHODS AND COMPOSITIONS FOR REDUCING PATHOGENIC ISOFORMS Public/Granted day:2023-03-16
Information query
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