Use of MIR-92A or MIR-145 in the treatment of Angelman syndrome
AssigneeOvid Therapeutics Inc.
InventorMatthew During
Life of this patent
Estimating the term…
Abstract
Expression of micoRNAs that negatively regulate the activity of the SNHG14 gene for can be used in treatment of Angelman Syndrome. Such microRNAs include, for example, MIR-92a and/or MIR-145, as well as analogues and variants thereof, for use in treatment of Angelman Syndrome. Expression vectors such as, for example, AAV vectors may be used to transduce cells for introduction of MIR-92a and/or MIR-145 into target tissues for treatment of Angelman Syndrome.
Claims29
A method for treating Angelman Syndrome in a patient in need thereof, comprising administering to the patient a vector including a nucleic acid encoding microRNA-145, pri-miR145, pre-miR145, microRNA-92a, pri-miR-92a or pre-miRNA-92a, wherein said nucleic acid is operably linked to a promoter, wherein one or more symptoms of the Angelman Syndrome are improved.
A method of treating Angelman Syndrome in a patient in need thereof comprising administering to the patient an effective amount of a pharmaceutical composition that increases the level of microRNA-145 and/or microRNA-92a molecules in the patient's brain, wherein the composition includes microRNA-145, pri-miR145, pre-miR145, microRNA-92a, pri-miR92a or pre-miR92a, or the composition includes a vector including nucleic acid encoding microRNA-145, pri-miR145, pre-miR145, microRNA-92a, pri-miR92a or pre-miR92a.
Dependencies are inferred from the claim text.