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:
Feb. 08, 2022
Filed:
Aug. 23, 2018
The Board of Trustees of the University of Illinois, Urbana, IL (US);
Yugang Bai, Changsha, CN;
Steven C. Zimmerman, Champaign, IL (US);
Auinash Kalsotra, Champaign, IL (US);
The Board of Trustees of the University of Illinois, Urbana, IL (US);
Abstract
Abnormally long r(CUG)n repeat expansion is believed to be the major cause of Myotonic dystrophy type 1 (DM1) because it binds to muscleblind-like 1 (MBNL 1) protein which regulates RNA splicing, leading to the mis-splicing of more than 100 pre-mRNAs. The rational design of oligomers with alternating bisamidine and melamine structure resulted in good binding affinity to the RNA target because of a multivalent effect. The oligomers also showed excellent activity in disrupting nuclear foci, reversing the mis-splicing of IR minigene, and sabotaging the toxic RNA biosynthesis. Excellent activity inbased DM1 models was also observed for the oligomers, rescuing the climbing ability of the flies upon oral treatment.