Inhibitors of cacna1a/alpha1a subunit internal ribosomal entry site (ires) and methods of treating spinocerebellar ataxia type 6
AssigneeThe University of Chicago
InventorsChristopher M. Gomez, Yu Miyazaki, Xiaofei Du
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Abstract
The invention provides methods of treating polyglutamine diseases, e.g., spinocerebellar ataxia Type 6, in a subject, comprising administering to the subject an IRES inhibitor in an amount effective for treating the SCA6 in the subject. Also provided herein are the IRES inhibitors, and pharmaceutical compositions comprising the same.
Claims29
A method of treating a subject with spinocerebellar ataxia Type 6 (SCA6) or with a predisposition to spinocerebellar ataxia Type 6 (SCA6), comprising the step of administering to the subject (i) an antisense molecule, (ii) a vector encoding the antisense molecule, (iii) a cell comprising the vector or antisense molecule, (iv) a extracellular vesicle comprising the antisense molecule, or (v) a combination thereof, wherein the antisense molecule binds to a portion of an IRES of a CACNA1A gene comprising the sequence of SEQ ID NO:
- 180.
(canceled)
5-13. (canceled)
A synthetic antisense molecule which specifically binds to a portion of an IRES of a CACNA1A gene comprising the sequence of SEQ ID NO:
- 180 comprising at least one non-naturally occurring nucleotide or at least one non-naturally occurring internucleotide linkage.
A recombinant expression vector comprising a nucleotide sequence encoding an antisense molecule which specifically binds to a portion of an IRES of a CACNA1A gene comprising the sequence of SEQ ID NO:
- 180.
(canceled)
21-22. (canceled)
24-25. (canceled)
27-29. (canceled)
31-32. (canceled)
A pharmaceutical composition comprising (i) an antisense molecule, (ii) a vector encoding the antisense molecule, (iii) a cell comprising the vector or antisense molecule, (iv) a extracellular vesicle comprising the antisense molecule, or (v) a combination thereof, wherein the antisense molecule binds to a portion of an IRES of a CACNA1A gene comprising the base sequence of SEQ ID NO:
- 180 or SEQ ID NO:
- 179, and a pharmaceutically acceptable carrier, diluent, or excipient, wherein the pharmaceutically acceptable carrier, diluent, or excipient is synthetic, when the antisense molecule, cell, or extracellular vesicle is naturally occurring.
34-39. (canceled)
A kit comprising (i) an antisense molecule, (ii) a vector encoding the antisense molecule, (iii) a cell comprising the vector or antisense molecule, (iv) a extracellular vesicle comprising the antisense molecule, or (v) a combination thereof, wherein the antisense molecule binds to a portion of an IRES of a CACNA1A gene comprising the base sequence of SEQ ID NO:
- 180 or SEQ ID NO:
- 179, (ii) and a device for administration to a subject.
41-46. (canceled)
Dependencies are inferred from the claim text.