Emory University

University of Florida Research Foundation Inc

Gary J Bassell

Xiaodi Yao

Christina Gross

Lyndon Lien

David B Rye

Andrew Jenkins

Maurice Scott Swanson

Eric Tzy-Shi Wang

Disclosed are methods of treating a disorder or disease associated with myotonic dystrophy. Methods of treating a CNS dysfunction and/or cognitive impairment associated with myotonic dystrophy in a subject comprising administering a therapeutically effective amount of a GABAA receptor antagonist or inverse agonist to the subject are disclosed. Methods of treating a myotonic dystrophy associated disease or disorder caused by mis-splicing of GABRG2 in a subject comprising administering a therapeutically effective amount of a GABAA receptor antagonist or inverse agonist to the subject are disclosed. Methods of improving cognitive function or alertness in a subject having myotonic dystrophy comprising administering a therapeutically effective amount of a GABAA receptor antagonist or inverse agonist to the subject are disclosed. Examples of the GABAA receptor antagonist or inverse agonist include flumazenil, clarithromycin, a fluoroquinolone, picrotoxin, bicuculline, gabazine, cicutoxin, oenan-thotoxin, pentylenetetrazol, Ro15-4513, sarmazenil, amentoflavone, zinc, and any combination thereof.

Treatment of conditions associated with myotonic dystrophy

This disclosure relates to manipulating microRNA for the management of neurological disorders and compositions related thereto. In certain embodiments, the disclosure contemplates inhibition of miR324 or miR324-5p, e.g., the use of nucleobase polymers for antisense disruptions or RNA interference of miR-324 expression or for miR324-5p binding in order to increase Kv4.2 expression. In certain embodiments, the disclosure relates to methods of treating or preventing a neurological disease or condition comprising administering an effective amount of an inhibitor to a subject in need thereto.

Manipulating microRNA for the management of neurological diseases or conditions and compositions related thereto

Growing community of inventors

Snellville, GA, United States of America

Gary J Bassell