Methods and pharmaceutical compositions for the treatment of cystic fibrosis
View Patent ↗The present invention relates to methods and pharmaceutical compositions for the treatment of cystic fibrosis. In particular, the present invention relates to a method of treating cystic fibrosis in a subject in need thereof comprising administering to the subject a therapeutically effective amount of a nucleic acid miR-9 inhibitor.
1. A method of treating cystic fibrosis in a subject in need thereof comprising administering to the subject a therapeutically effective amount of a nucleic acid miR-9 inhibitor comprising the nucleic acid sequence of SEQ ID NO: 3, wherein the nucleic acid miR-9 inhibitor does not bind directly to miR-9 but instead binds to a miR-9 mRNA target site in an anoctamin-1 (ANO1) nucleic acid sequence.
2. The method of claim 1 wherein the miR-9 mRNA target site is located on a ANO1 3′UTR.
3. The method of claim 1 wherein the nucleic acid miR-9 inhibitor is a Target Site Blocker (TSB).
4. The method of claim 1 wherein the nucleic acid miR-9 inhibitor comprises LNA nucleotides, or morpholino nucleotides, or 2′-O-methyl modified nucleotides, or 2′-O-methoxyethyl modified nucleotides, or 2′-fluro modified nucleotides.
5. The method of claim 1 wherein the nucleic acid miR-9 inhibitor is delivered using a viral vector.
6. The method of claim 5 wherein the vector is an AAV vector.
7. The method of claim 1 wherein the nucleic acid miR-9 inhibitor is administered to the subject using a method that enables the nucleic acid miR-9 inhibitor to reach the lungs.
8. A viral vector encoding a nucleic acid miR-9 inhibitor comprising the sequence of SEQ ID NO: 3 wherein the nucleic acid miR-9 inhibitor is not configured to bind directly to miR-9 but instead is configured to bind to a miR-9 mRNA target site in an anoctamin-1 (ANO1) nucleic acid sequence.
9. A composition comprising a viral vector encoding a nucleic acid miR-9 inhibitor comprising the sequence of SEQ ID NO: 3 wherein the nucleic acid miR-9 inhibitor is not configured to bind directly to miR-9 but instead is configured to bind to a miR-9 mRNA target site in an anoctamin-1 (ANO1) nucleic acid sequence; and a pharmaceutically acceptable carrier.