Patent ID: 11932852
Assignee: BENITEC IP HOLDINGS INC.
Field: Pharmaceuticals (Chemistry)
Classification: CPC C  A | IPC C

Claim 18:
19. A method of treating oculopharyngeal muscular dystrophy (OPMD) in a subject suffering therefrom, said method comprising administering to the subject:
(a) the nucleic acid according to claim 1;
(b) a ddRNAi construct comprising the nucleic acid of (a);
(c) a DNA construct comprising a ddRNAi construct of (b) and a PABPN1 construct comprising a DNA sequence encoding a functional PABPN1 protein having a mRNA transcript which is not targeted by the shmiR(s) encoded by the ddRNAi construct;
(d) an expression vector comprising (a), (b) or (c);
(e) a plurality of expression vectors comprising at least one expression vector comprising the ddRNAi construct of (b) and at least one expression vector comprising a PABPN1 construct comprising a DNA sequence encoding a functional PABPN1 protein having a mRNA transcript which is not targeted by the shmiR(s) encoded by the ddRNAi construct; or
(f) a composition comprising any one of (a)-(e).