Invention Grant
- Patent Title: Recombinant virus products and methods for inhibition of expression of myotilin
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Application No.: US14853517Application Date: 2015-09-14
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Publication No.: US10196636B2Publication Date: 2019-02-05
- Inventor: Scott Quenton Harper , Jian Liu
- Applicant: NATIONWIDE CHILDREN'S HOSPITAL, INC.
- Applicant Address: US OH Columbus
- Assignee: NATIONWIDE CHILDREN'S HOSPITAL, INC.
- Current Assignee: NATIONWIDE CHILDREN'S HOSPITAL, INC.
- Current Assignee Address: US OH Columbus
- Agency: Marshall, Gerstein & Borun LLP
- Main IPC: C12N15/86
- IPC: C12N15/86 ; C12N15/113 ; C07K14/47
Abstract:
The present invention relates to RNA interference-based methods for inhibiting the expression of the myotilin gene. Recombinant adeno-associated viruses of the invention deliver DNAs encoding microRNAs that knock down the expression of myotilin. The methods have application in the treatment of muscular dystrophies such as Limb Girdle Muscular Dystrophy Type 1A.
Public/Granted literature
- US20160310548A1 RECOMBINANT VIRUS PRODUCTS AND METHODS FOR INHIBITION OF EXPRESSION OF MYOTILIN Public/Granted day:2016-10-27
Information query
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