Methods and agents to increase therapeutic dystrophin expression in muscle
AssigneeReveraGen BioPharma, Inc.
InventorsEric Hoffman, Alyson Fiorillo
Life of this patent
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Abstract
Agents and methods for increasing dystrophin protein expression in muscle through blocking of specific microRNAs and microRNA binding sites on the dystrophin 3′ untranslated region (miR-146a, miRNA-146b, miR-223, miR-320a, miR374a, and miR-382). Methods for increasing the amount of dystrophin useful for effective therapeutic intervention for Becker muscular dystrophy, Duchenne muscular dystrophy, and other disorders where loss of dystrophin from muscle causes pathology.
Claims39
A nucleic acid molecule or derivative thereof that binds to at least one of miR-146a, miR-146b-5p, miR-223, miR-320a, miR-374a, or miR-382 or to portions thereof, and that increases expression of dystrophin protein.
A nucleic acid molecule or derivative thereof able to functionally inactivate the action of miR-146a, miRNA-146b-5p, miR-223, miR-320a, miR374a, and/or miR-382 and increase dystrophin protein translation in muscle.
Dependencies are inferred from the claim text.