The patent badge is an abbreviated version of the USPTO patent document. The patent badge does contain a link to the full patent document.
The patent badge is an abbreviated version of the USPTO patent document. The patent badge covers the following: Patent number, Date patent was issued, Date patent was filed, Title of the patent, Applicant, Inventor, Assignee, Attorney firm, Primary examiner, Assistant examiner, CPCs, and Abstract. The patent badge does contain a link to the full patent document (in Adobe Acrobat format, aka pdf). To download or print any patent click here.
Patent No.:
Date of Patent:
Apr. 30, 2024
Filed:
Mar. 23, 2018
The University Court of the University of Edinburgh, Edinburgh, GB;
The University Court of the University of Glasgow, Glasgow, GB;
Adrian Bird, Edinburgh, GB;
Rebekah Tillotson, Edinburgh, GB;
Stuart Robert Cobb, Edinburgh, GB;
Ralph David Hector, Edinburgh, GB;
THE UNIVERSITY COURT OF THE UNIVERSITY OF EDINBURGH, Edinburgh, GB;
THE UNIVERSITY COURT OF THE UNIVERSITY OF GLASGOW, Glasgow, GB;
Abstract
The present invention provides nucleic acid molecules comprising a MeCP2 expression cassette, the expression cassette comprising, in operable linkage from 5′ to 3′: a 5′ transcriptional control region comprising a promoter capable of driving transcription in neural cells; an open reading frame encoding a MeCP2 protein; translation control signals; a 3′ untranslated region (3′UTR) comprising one or more of: (i) a binding site for mir-22; (ii) a binding site for mir-19; (iii) a binding site for miR-132; (iv) a binding site for miR124; and (v) an AU-rich element; and transcriptional termination signals; wherein the MeCP2 expression cassette is not more than about 5 kb in length. The invention further provides viral vectors, especially vectors derived from adeno-associated virus (AAV), for use in therapeutic delivery of such expression cassettes. The nucleic acid molecules and viral vectors disclosed herein provide novel tools for expressing MeCP2 and are of particular value in the treatment of disorders associated with reduced MeCP2 activity, including Rett syndrome.