Methods for modulating expression of c9orf72 antisense transcript
AssigneesIonis Pharmaceuticals, Inc., The Regents of the University of California, Ludwig Institute for Cancer Research
InventorsC. Frank Bennett, Frank Rigo, Don W. Cleveland, Clotilde Lagier-Tourenne, John M. Ravits, Michael W. Baughn
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Abstract
Disclosed herein are methods for reducing expression of C90RF72 antisense transcript in an animal with C90RF72 antisense transcript specific inhibitors. Such methods are useful to treat, prevent, or ameliorate neurodegenerative diseases in an individual in need thereof. Such C90RF72 antisense transcript specific inhibitors include antisense compounds.
Claims51
A method, comprising contacting a cell with a C9ORF72 antisense transcript specific inhibitor.
A method, comprising contacting a cell with a C9ORF72 antisense transcript specific inhibitor and a C9ORF72 sense transcript specific inhibitor.
A method, comprising contacting a cell with a C9ORF72 antisense transcript specific inhibitor;
- and thereby reducing the level or expression of C9ORF72 antisense transcript in the cell.
A method, comprising contacting a cell with a C9ORF72 antisense transcript specific inhibitor and a C9ORF72 sense transcript specific inhibitor;
- and thereby reducing the level or expression of both C9ORF72 antisense transcript and C9ORF72 sense transcript in the cell.
A method, comprising administering to an animal in need thereof a therapeutically effective amount of a C9ORF72 antisense transcript specific inhibitor.
A method, comprising coadministering to an animal in need thereof a therapeutically effective amount of a C9ORF72 antisense transcript inhibitor and a therapeutically effective amount of a C9ORF72 sense transcript inhibitor.
A method, comprising:
- identifying an animal having a C9ORF72 associated disease;
- and
- administering to the animal a therapeutically effective amount of a C9ORF72 antisense transcript specific inhibitor.
A method, comprising:
- identifying an animal having a C9ORF72 associated disease;
- and
- coadministering to the animal a therapeutically effective amount of a C9ORF72 antisense transcript specific inhibitor and a therapeutically effective amount of a C9ORF72 sense transcript inhibitor.
The method of any preceding claim, wherein the C9ORF72 antisense transcript specific antisense compound is at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% complementary to a C9ORF72 antisense transcript.
The method of any preceding claim, wherein the C9ORF72 sense transcript specific antisense compound is at least 80%, at least 85%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% complementary to a C9ORF72 sense transcript.
The method of any preceding claim, wherein the C9ORF72 antisense transcript is SEQ ID NO:
- 11.
The method of any preceding claim, wherein the C9ORF72 sense transcript is any of SEQ ID NO:
- 1-10.
The method of any preceding claim, wherein the contacting or administering reduces C9ORF72 foci.
The method of any preceding claim, wherein the contacting or administering reduces C9ORF72 antisense transcript associated RAN translation products.
The method of claim 33, wherein the C9ORF72 antisense transcript associated RAN translation products are any of poly-(proline-alanine), poly-(proline-arginine), and poly-(proline-glycine).
The method of claim 35, wherein the parental administration is any of injection or infusion.
The method of any preceding claim, wherein the C9ORF72 antisense transcript specific antisense compound is an antisense oligonucleotide.
The method of any preceding claim, wherein the C9ORF72 sense transcript specific antisense compound is an antisense oligonucleotide.
The method of any preceding claim, wherein the antisense oligonucleotide is a gapmer.
Dependencies are inferred from the claim text.