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:
Jun. 28, 2016
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 rationally designed multi-targeting therapeutic agents for myotonic dystrophy type 1 (DM1), an incurable neuromuscular disease that originates in an abnormal expansion of CTG repeats (CTG) in the DMPK gene. The rationally designed small molecules target the DM1 pathobiology in three distinct ways: (1) binding the expanded trinucleotide repeat, CTG, and inhibiting its transcription to the toxic CUGRNA, (2) binding the CUGRNA and releasing sequestered muscleblind-like protein (MBNL1), and (3) cleaving the toxic CUGin an RNase-like manner. Importantly, the compounds can reduce the levels of CUGin DM1 model cells and reverse two separate CUG-induced phenotypes of DM1.