The patent badge is an abbreviated version of the USPTO patent document. The patent badge does contain a link to the full patent document.
The patent badge is an abbreviated version of the USPTO patent document. The patent badge covers the following: Patent number, Date patent was issued, Date patent was filed, Title of the patent, Applicant, Inventor, Assignee, Attorney firm, Primary examiner, Assistant examiner, CPCs, and Abstract. The patent badge does contain a link to the full patent document (in Adobe Acrobat format, aka pdf). To download or print any patent click here.
Patent No.:
Date of Patent:
Apr. 23, 2019
Filed:
Aug. 10, 2015
The Board of Trustees of the University of Illinois, Urbana, IL (US);
Steven C. Zimmerman, Champaign, IL (US);
Long M. Luu, Urbana, IL (US);
Lien T. T. Nguyen, Urbana, IL (US);
The Board of Trustees of the University of Illinois, Urbana, IL (US);
Abstract
The invention provides compounds, compositions and therapeutic methods. The compounds and compositions can be used for the treatment of myotonic dystrophy. The compounds can selectively bind to CUG repeats in RNA, or to CTG repeats in DNA, and inhibit replication of the nucleic acids. RNA-targeted therapeutic agents for the treatment of myotonic dystrophy type 1 (DM1) are described. In one embodiment, two bisamidinium ligands are linked using 'click' chemistry to form a heterodimer that is a potent inhibitor of the MBNL1-rCUGcomplex (K=25±8 nM), is relatively non-toxic to HeLa cells, dissolves nuclear foci, corrects >80% of the IR misregulated alternative splicing in DM1 model cells (1 μM), and shows improvement of disease phenotypes in a DM1model.