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

Claim 0:
1. A nucleic acid comprising a DNA sequence which encodes a short hairpin micro-RNA (shmiR) targeting a transcript of PABPN1, said shmiR comprising:
an effector sequence of at least 17 nucleotides in length;
an effector complement sequence;
a stemloop sequence; and
a primary micro RNA (pri-miRNA) backbone;
wherein the effector sequence is complementary to a region of corresponding length in an RNA transcript set forth in SEQ ID NO: 9 or complementary to a region of corresponding length in an RNA transcript set forth in SEQ ID NO: 9 with the exception of 1, 2, 3 or 4 base mismatches relative to the RNA transcript set forth in SEQ ID NO: 9.