Methods and sequences to suppress primate huntington gene expression in vivo
First Claim
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1. An isolated nucleic acid duplex comprising a first strand of nucleic acid and a second strand of nucleic acid, wherein the first strand comprises at least 19 contiguous nucleotides encoded by the group consisting of SEQ ID NO:
- 1, and SEQ ID NO;
4 and wherein the second strand is complementary to at least 15 contiguous nucleotides of the first strand.
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Abstract
Disclosed herein are sequences, molecules and methods used to suppress the expression of HD genes encoding for huntingtin protein in primates including Macaca mulatta and Homo sapiens. These sequences, molecules and methods aid in the study of the pathogenesis of HD and can also provide a treatment for this disease by reducing HD mRNA without causing death, locomotor impairment or cellular alterations of the Macaca mulatta and Homo sapiens.
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39 Claims
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1. An isolated nucleic acid duplex comprising a first strand of nucleic acid and a second strand of nucleic acid, wherein the first strand comprises at least 19 contiguous nucleotides encoded by the group consisting of SEQ ID NO:
- 1, and SEQ ID NO;
4 and wherein the second strand is complementary to at least 15 contiguous nucleotides of the first strand. - View Dependent Claims (2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16, 17, 18, 19, 20, 21, 22, 23, 24, 25, 26, 27, 28, 29, 30, 31, 32, 33, 34, 35, 36, 37, 38, 39)
- 1, and SEQ ID NO;
Specification