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AssigneeThe Research Foundation for The State University of New York
InventorsHormoz Mazdiyasni, John Andrew Berglund
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The present disclosure provides compositions that inhibit microsatellite promoted expression of deleterious expansions by targeting expanded CTG DNA as well as methods using such compositions for use in treating or ameliorating the effects of a medical condition involving trinucleotide repeats in a subject. In embodiments, the present disclosure provides a method for treating a medical condition involving trinucleotide repeats, including a disease such as DM1, in a subject in need thereof by administering to the subject an effective amount of a modified polycyclic compound of the present disclosure. Processes for synthesizing modified polycyclic compounds that rescue mis-splicing at low nanomolar concentration with negligible toxicity are also disclosed.
A method of treating a medical condition involving expanded CTG and/or CCTG repeats in a subject in need thereof, said method comprising, administering to the subject an effective amount of at least one polycyclic compound selected from the group consisting of
A method of treating a medical condition involving expanded CTG repeats in a subject in need thereof, said method comprising, administering to the subject an effective amount of at least one polycyclic compound selected from the group consisting of:
Dependencies are inferred from the claim text.