University of Rochester

University of Florida Research Foundation Inc

Ionis Pharmaceuticals, Inc.

Charles A Thornton

Robert Osborne

Maurice Scott Swanson

Thurman Wheeler

Krzysztof Sobczak

Susan M Freier

C Frank Bennett

Seng H Cheng

Benjamin L Miller

Robert A MacLeod

Brian R McNaughton

Sanjay K Pandey

Bruce M Wentworth

Joseph E Wedekind

Peter C Gareiss

Andrew Leger

Rahul N Kanadia

Jill Miller

Maurice S Swanson

Krzysztof Sobezak

Maurice Swanson

Sanjey K Pandey

Provided herein are methods, compounds, and compositions for reducing expression of a DMPK mRNA and protein in an animal. Also provided herein are methods, compounds, and compositions for preferentially reducing CUGexp DMPK RNA, reducing myotonia or reducing spliceopathy in an animal. Such methods, compounds, and compositions are useful to treat, prevent, delay, or ameliorate type 1 myotonic dystrophy, or a symptom thereof.

Modulation of dystrophia myotonica-protein kinase (DMPK) expression

Disclosed are compositions and methods related to the interaction of polyCUG and polyCCUG repeat RNA and proteins that bind to these repetitive RNA sequences. Also disclosed are methods of treating DM1 or DM2 comprising inhibiting the interaction of poly(CUG)or poly(CCUG)RNA with muscleblind proteins, or by causing improvement of spliceopathy in myotonic dystrophy.

Compositions and methods related to protein displacement therapy for myotonic distrophy

The present invention relates to homo- and hetero-dimer compounds formed by a disulfide, sulfinyl thio, or olefin bond between two monomers. A method of making a homo- or hetero-dimer compound is also disclosed. The present invention also relates to monomer compounds capable of forming homo- or hetero-dimer compounds, as well as oligomers formed via linkage of one or more dimers. Also disclosed are methods of inhibiting the activity of target RNA molecules, particularly those having a secondary structure that include a stem or stem-loop formation. Dimer compounds capable of inhibiting the activity of an HIV-I RNA frameshifting stem-loop and a (CUG)n expanded repeat stem-loop are disclosed, as are methods of treating diseases associated with these target RNA molecules. The dimer compounds can also be used for selectively detecting presence of the target RNA molecule in a sample.

Nucleic acid binding compounds and methods of use

The present invention provides methods and compositions for the treatment of diseases associated with aberrant microsatellite expansions. Methods of the present invention comprise the use of recombinant adeno-associated virus vectors containing a transgene encoding at least one muscleblind protein. The present invention also provides an animal model for a disease associated with aberrant microsatellite expansion.

Methods and compositions for treatment of myotonia

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Rochester, NY, United States of America

Charles A Thornton