Patent ID: 11969479
Assignee: THE UNIVERSITY COURT OF THE UNIVERSITY OF GLASGOW
Field: Pharmaceuticals (Chemistry)
Classification: CPC A  C | IPC A  C

Claim 0:
1. A nucleic acid molecule comprising a MeCP2 expression cassette, the expression cassette comprising, in operable linkage from 5′ to 3′:
(a) a 5′ transcriptional control region comprising a promoter capable of driving transcription in neural cells, wherein the promoter is a MeCP2 promoter comprising a MeCP2 silencer element and a CNS regulatory element;
(b) a Kozak sequence;
(c) an open reading frame encoding a MeCP2 protein;
(d) a 3′ untranslated region (3′UTR) comprising a MeCP2 polyadenylation signal
a binding site for miR-22,
a binding site for miR-19;
a binding site for miR-132,
a binding site for miR-124, and
one or more AU-rich element of the sequence AUUUA; and
(e) transcriptional termination signals; and, wherein the MeCP2 expression cassette is not more than about 5 kb in length.