Invention Grant
- Patent Title: Gapmers and methods of using the same for the treatment of muscular dystrophy
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Application No.: US16649122Application Date: 2018-09-19
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Publication No.: US11518995B2Publication Date: 2022-12-06
- Inventor: Yi-wen Chen , Toshifumi Yokota , Rika Yokota-Maruyama , Yusuke Echigoya
- Applicant: Children's National Medical Center , The Governors of the University of Alberta
- Applicant Address: US DC Washington; CA Edmonton
- Assignee: Children's National Medical Center,The Governors of the University of Alberta
- Current Assignee: Children's National Medical Center,The Governors of the University of Alberta
- Current Assignee Address: US DC Washington; CA Edmonton
- Agency: Oblon, McClelland, Maier & Neustadt, L.L.P.
- International Application: PCT/US2018/051776 WO 20180919
- International Announcement: WO2019/060432 WO 20190328
- Main IPC: C12N15/11
- IPC: C12N15/11 ; C12N15/113
Abstract:
The disclosure relates to compositions comprising a nucleotide sequence having two domains: a locked nucleic acid (LNA) domain and a DNA gap domain, wherein nucleotide sequence binds to an endogenous DUX4 mRNA sequence disrupts DUX4 expression.
Public/Granted literature
- US20200291398A1 GAPMERS AND METHODS OF USING THE SAME FOR THE TREATMENT OF MUSCULAR DYSTROPHY Public/Granted day:2020-09-17
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