- Patent Title: Compositions and methods for treating amyotrophic lateral sclerosis
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Application No.: US15026774Application Date: 2014-10-03
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Publication No.: US10174328B2Publication Date: 2019-01-08
- Inventor: Arthur M. Krieg , James Barsoum
- Applicant: Translate Bio MA, Inc.
- Applicant Address: US MA Lexington
- Assignee: Translate Bio MA, Inc.
- Current Assignee: Translate Bio MA, Inc.
- Current Assignee Address: US MA Lexington
- Agency: Wolf, Greenfield & Sacks, P.C.
- International Application: PCT/US2014/059111 WO 20141003
- International Announcement: WO2015/051283 WO 20150409
- Main IPC: C12N15/11
- IPC: C12N15/11 ; C12N15/113 ; A61K31/7088 ; A61K31/711 ; A61K31/7105 ; A61K31/712 ; A61K31/713 ; C12N15/63 ; A61K48/00 ; A61K9/00 ; C12N15/67

Abstract:
Methods for modulating expression of SMN1 and/or SMN2 in cells obtained from subjects having ALS or in subjects having ALS using single stranded oligonucleotides are provided. Methods for treating ALS using single stranded oligonucleotides are also provided.
Public/Granted literature
- US20160222391A1 COMPOSITIONS AND METHODS FOR TREATING AMYOTROPHIC LATERAL SCLEROSIS Public/Granted day:2016-08-04
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