Invention Grant
- Patent Title: Induced exon inclusion in spinal muscle atrophy
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Application No.: US14360895Application Date: 2012-11-30
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Publication No.: US09944926B2Publication Date: 2018-04-17
- Inventor: Peter Linsley , Brian James Leppert
- Applicant: Sarepta Therapeutics, Inc.
- Applicant Address: US WA Bothell
- Assignee: Sarepta Therapeutics, Inc.
- Current Assignee: Sarepta Therapeutics, Inc.
- Current Assignee Address: US WA Bothell
- Agency: Finnegan, Henderson, Farabow, Garrett & Dunner, LLP
- International Application: PCT/US2012/067475 WO 20121130
- International Announcement: WO2013/082551 WO 20130606
- Main IPC: C12N15/113
- IPC: C12N15/113 ; C12N15/11
Abstract:
The invention relates to the use of an antisense compound for inducing exon inclusion as a treatment for Spinal Muscle Atrophy (SMA). More particularly it relates to inducing inclusion of exon 7 to restore levels of Survival Motor Neuron (SMN) protein encoded by the Survival Motor Neuron (SMN) gene.
Public/Granted literature
- US20140329772A1 INDUCED EXON INCLUSION IN SPINAL MUSCLE ATROPHY Public/Granted day:2014-11-06
Information query
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