Invention Grant
- Patent Title: Recombinant adeno-associated virus delivery of exon 2-targeted U7SNRNA polynucleotide constructs
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Application No.: US14785769Application Date: 2014-04-18
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Publication No.: US09862945B2Publication Date: 2018-01-09
- Inventor: Kevin Flanigan , Adeline Vulin-Chaffiol , Nicolas Wein
- 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/US2014/034702 WO 20140418
- International Announcement: WO2014/172669 WO 20141023
- Main IPC: C07H21/02
- IPC: C07H21/02 ; A61K48/00 ; C12N15/113 ; C07K14/47 ; C12N15/86 ; C12N15/11 ; C12N7/00 ; A01K67/00
Abstract:
The present invention relates to recombinant adeno-associated virus (rAAV) delivery of polynucleotides for treating Duchenne Muscular Dystrophy resulting from the duplication of DMD exon 2. The invention provides rAAV products and methods of using the rAAV in the treatment of Duchenne Muscular Dystrophy.
Public/Granted literature
- US20160076028A1 RECOMBINANT ADENO-ASSOCIATED VIRUS DELIVERY OF EXON 2-TARGETED U7SNRNA POLYNUCLEOTIDE CONSTRUCTS Public/Granted day:2016-03-17
Information query
