TARGET SEQUENCES AND METHODS TO IDENTIFY THE SAME, USEFUL IN TREATMENT OF NEURODEGENERATIVE DISEASES
First Claim
1. A method for identifying a compound that modulates cell death, said method comprising:
- a) contacting a compound with a polypeptide comprising an amino acid sequence selected from the group consisting of SEQ ID NO;
46, 47, 49, 51-60, 62-67, 69, 71, 75-82 and 85-90; and
b) determining the binding affinity of the compound to the polypeptide.
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Accused Products
Abstract
The present invention relates to methods and assays for identifying agents capable of inhibiting the mutant huntingtin protein, inhibiting or reducing cell death, in particular cell death associated with polyglutamine-induced protein aggregation, which inhibition is useful in the prevention, amelioration and/or treatment of neurodegenerative diseases, and Huntington'"'"'s disease more generally. In particular, the present invention provides methods and assays for identifying agents for use in the prevention and/or treatment of Huntingtons disease. The invention provides polypeptide and nucleic acid TARGETs and siRNA sequences based on these TARGETS.
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Citations
34 Claims
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1. A method for identifying a compound that modulates cell death, said method comprising:
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a) contacting a compound with a polypeptide comprising an amino acid sequence selected from the group consisting of SEQ ID NO;
46, 47, 49, 51-60, 62-67, 69, 71, 75-82 and 85-90; andb) determining the binding affinity of the compound to the polypeptide. - View Dependent Claims (2, 5, 6, 7, 8, 9, 10, 11)
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3. A method for identifying a compound that modulates cell death, said method comprising:
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a) contacting a compound with a polypeptide comprising an amino acid sequence selected from the group consisting of SEQ ID NO;
46, 47, 49, 51-60, 62-67, 69, 71, 75-82 and 85-90; andb) determining the ability of the compound inhibit the expression or activity of the polypeptide. - View Dependent Claims (4)
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12. An agent effective in modulating polyglutamine-induced cell death, selected from the group consisting of an antisense polynucleotide, a ribozyme, and a small interfering RNA (siRNA), wherein said agent comprises a nucleic acid sequence complementary to, or engineered from, a naturally-occurring polynucleotide sequence of about 17 to about 30 contiguous nucleotides of a nucleic acid sequence selected from the group consisting of SEQ ID NO:
- 1, 2, 4, 6-15, 17-22, 24, 26, 30-37, 40-45.
- View Dependent Claims (13, 14, 15, 16, 17, 18, 19, 20, 21, 22, 23, 24, 25, 26)
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Specification