Invention Grant
- Patent Title: Methods for treating progeroid laminopathies using oligonucleotide analogues targeting human LMNA
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Application No.: US15084255Application Date: 2016-03-29
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Publication No.: US09833468B2Publication Date: 2017-12-05
- Inventor: Ryszard Kole , Francis S. Collins , Michael R. Erdos , Kan Cao
- Applicant: Sarepta Therapeutics, Inc. , The United States of America, as represented by the Secretary, Dept. of Health and Human Services , University of Maryland
- Applicant Address: US MA Cambridge US DC Washington US MD College Park
- Assignee: Sarepta Therapeutics, Inc.,The United States of America, as represneted by the Secretary, Dept. of Health and Human Services,University of Maryland
- Current Assignee: Sarepta Therapeutics, Inc.,The United States of America, as represneted by the Secretary, Dept. of Health and Human Services,University of Maryland
- Current Assignee Address: US MA Cambridge US DC Washington US MD College Park
- Agency: Seed IP Law Group LLP
- Main IPC: A61K31/7125
- IPC: A61K31/7125 ; C12N15/113 ; A61K31/712 ; A61K47/42 ; A61K47/48 ; A61K31/713 ; C12N15/11

Abstract:
Provided are methods of treatment in subjects having progeroid diseases and related conditions which rely upon LMNA-targeted antisense oligonucleotides for reducing expression of one or more aberrantly spliced LMNA mRNA isoforms that encode progerin.
Public/Granted literature
- US20170051278A1 METHODS FOR TREATING PROGEROID LAMINOPATHIES USING OLIGONUCLEOTIDE ANALOGUES TARGETING HUMAN LMNA Public/Granted day:2017-02-23
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