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 (CTGexp) in the DMPK gene. The rationally designed small molecules target the DM1 pathobiology in three distinct ways: (1) binding the expanded trinucleotide repeat, CTGexp, and inhibiting its transcription to the toxic CUGexp RNA, (2) binding the CUGexp RNA and releasing sequestered muscleblind-like protein (MBNL1), and (3) cleaving the toxic CUGexp in an RNase-like manner. Importantly, the compounds can reduce the levels of CUGexp in DM1 model cells and reverse two separate CUGexp-induced phenotypes of DM1.
Type:
Application
Filed:
August 10, 2015
Publication date:
February 25, 2016
Applicant:
The Boad of Trustees of the University of Illinois
Inventors:
Steven C. Zimmerman, Long M. Luu, Lien T.T. Nguyen
Abstract: The present invention discloses methods and materials for delivering a cargo compound into a cancer cell. Delivery of the cargo compound is accomplished by the use of protein transduction domains derived from cupredoxins. The invention further discloses methods for treating cancer and diagnosing cancer.
Type:
Application
Filed:
September 9, 2013
Publication date:
February 6, 2014
Applicant:
The Boad of Trustees of the University of Illinois
Inventors:
Ananda Chakrabarty, Tapas Das Gupta, Tohru Yamada, Arsenio Fialho