Invention Grant
- Patent Title: Adeno-associated virus vector delivery of microRNA-29 and micro-dystrophin to treat muscular dystrophy
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Application No.: US16092706Application Date: 2017-04-14
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Publication No.: US11406717B2Publication Date: 2022-08-09
- Inventor: Louise Rodino-Klapac , Jerry R. Mendell , Kristin N. Heller
- Applicant: RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL
- Applicant Address: US OH Columbus
- Assignee: RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL
- Current Assignee: RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL
- Current Assignee Address: US OH Columbus
- Agency: Marshall, Gerstein & Borun LLP
- International Application: PCT/US2017/027635 WO 20170414
- International Announcement: WO2017/181014 WO 20171019
- Main IPC: C07H21/04
- IPC: C07H21/04 ; A61K48/00 ; C07K14/47 ; A61K38/17 ; A61K31/7088 ; C12N15/11 ; C12N15/113 ; C12N15/86 ; A61P21/00 ; A61K9/00 ; A61P19/04 ; A61K35/761 ; C12N15/864
Abstract:
The invention provides for recombinant AAV vectors comprising a polynucleotide sequence comprising the guide strand of miR-29c and methods of using the recombinant vectors to reduce or prevent fibrosis in subjects suffering from dystrophinopathy or muscular dystrophy. The invention also provides for combination therapies comprising expressing both miR-29 and micro-dystrophin to reduce and prevent fibrosis in patients suffering from dystrophinopathy or muscular dystrophy.
Public/Granted literature
- US20190060489A1 ADENO-ASSOCIATED VIRUS VECTOR DELIVERY OF MICRORNA-29 AND MICRO-DYSTROPHIN TO TREAT MUSCULAR DYSTROPHY Public/Granted day:2019-02-28
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