CHARLES A THORNTON, MD
Psychiatric at Elmwood Ave, Rochester, NY

2010026, Oct 21, 2010

Jul 28, 2008

12/670772

Benjamin L. Miller - Penfield NY, US
Brian R. McNaughton - Rochester NY, US
Peter C. Gareiss - Rochester NY, US
Joseph Wedekind - Rochester NY, US
Charles Thornton - Rochester NY, US
Krzysztof Sobczak - Rochester NY, US

UNIVERSITY OF ROCHESTER - Rochester NY

A61K 9/127, C07K 5/10, C07K 5/08, C07K 5/06, A61K 38/05, C12Q 1/68, A61K 38/06, A61K 38/07, A61K 38/08, C12Q 1/70, C07H 21/02

424450, 530331, 530330, 514 38, 435 6, 514 219, 514 2191, 435 5, 536 231

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.

2013023, Sep 12, 2013

Jul 19, 2011

13/811181

C. Frank Bennett - Carlsbad CA, US
Susan M. Freier - San Diego CA, US
Robert A. MacLeod - San Diego CA, US
Sanjay K. Pandey - Encinitas CA, US
Charles A. Thornton - Rochester NY, US
Thurman Wheeler - Rochester NY, US
Seng H. Cheng - Natick MA, US
Andrew Leger - Boston MA, US
Bruce M. Wentworth - Northborough MA, US

UNIVERSITY OF ROCHESTER - Rochester NY
ISIS PHARMACEUTICALS, INC. - Carlsbad CA

C12N 15/113

514 44 A, 536 245

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.

7964570, Jun 21, 2011

Mar 10, 2005

10/591883

Maurice S. Swanson - Gainesville FL, US
Rahul N. Kanadia - Gainesville FL, US
Charles A. Thornton - Rochester NY, US

University of Florida Research Foundation, Inc. - Gainesville FL

A61K 48/00, C12N 5/00, C12N 5/02, C07H 21/02

514 44R, 435325, 536 231

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.

2010019, Jul 29, 2010

Sep 21, 2007

12/442354

Charles A. Thornton - Rochester NY, US
Thurman Wheeler - Rochester NY, US
Krzysztof Sobezak - Rochester NY, US
Robert Osborne - Rochester NY, US
Jill Miller - Rockport NY, US
Maurice Swanson - Gainesville FL, US

University of Rochester - Rochester NY
University of Florida Research Foundation, Inc. - Gainesville FL

A61K 38/14, C12Q 1/68, A61K 31/7088, C12N 5/00, C07H 21/00

514 8, 435 6, 514 44 A, 435325, 536 245

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.