Genetic and Pharmacological Transcriptional Upregulation of the Repressed FXN Gene as a Therapeutic Strategy for Friedreich Ataxia
AssigneeUniversity of Massachusetts
InventorsMichael R. Green, Minggang Fang
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Abstract
Methods of treating subjects having disorders associated with mutations in the FXN gene and/or having reduced expression of frataxin protein, e.g., Friedreich ataxia (FA). Generally, the methods include administering a therapeutically effective amount of an agent that increases expression of frataxin protein as described herein, e.g., an inhibitor of a FXN Repressing Factor (FXN-RF).
Claims8
A method of treating a subject having a disorder associated with a mutation in the FXN gene and having reduced expression of frataxin protein, the method comprising administering to the subject a therapeutically effective amount of a small molecule inhibitor of a FXN Repressing Factor (FXN-RF) selected from the group consisting of EZH2, EED, DNMT3A, DNMT3B, SETDB1, EHMT2/G9a, RING1B/RNF2, KDM5A, KDM5D, HDAC2, HDAC5, and SIRT7.
A method of treating a subject having a disorder associated with a mutation in the FXN gene and having reduced expression of frataxin protein, the method comprising administering to the subject a therapeutically effective amount of an inhibitory nucleic acid that targets and specifically reduces expression of a FXN Repressing Factor (FXN-RF) selected from the group consisting of EZH2, EED, DNMT3A, DNMT3B, SETDB1, EHMT2/G9a, RING1B/RNF2, KDM5A, KDM5D, HDAC2, HDAC5, and SIRT7.
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Dependencies are inferred from the claim text.