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US 2020/0392500 A1Published application
Aav-based treatment of cholesterol-related disorders
AssigneeUniversity of Massachusetts
InventorsGuangping Gao, Phillip D. Zamore, Jun Xie
Life of this patent
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Abstract
The invention in some aspects relates to methods and compositions for assessing the effectiveness of miRNA inhibitors. In other aspects of the invention, methods and compositions for treating cholesterol related disorders are provided. In one aspect of the invention, miRNA inhibitors against miR-122 and rAAV-based compositions comprising the same are provided.
Claims24
-56. (canceled)
A miRNA inhibitor comprising:
- a first miRNA binding site and a second miRNA binding site, wherein a first stem sequence flanks the first miRNA binding site at its 5′-end, a second stem sequence flanks the first miRNA binding site at its 3′-end and the second miRNA binding site at its 5′-end, and a third stem sequence flanks the second miRNA binding site at its 3′-end, wherein at least one of the miRNA binding sites comprises a nucleotide sequence complementary to a consensus sequence of the plurality of target miRNAs.
-67. (canceled)
(canceled)
A method, comprising
- obtaining a consensus sequence from a plurality of miRNAs, and
- generating a miRNA inhibitor targeting a plurality of miRNAs, wherein the miRNA inhibitor comprises a first miRNA binding site and a second miRNA binding site, wherein a first stem sequence flanks the first miRNA binding site at its 5′-end, a second stem sequence flanks the first miRNA binding site at its 3′-end and the second miRNA binding site at its 5′-end, and a third stem sequence flanks the second miRNA binding site at its 3′-end, wherein the miRNA inhibitor comprises a nucleotide sequence complementary to the consensus sequence of the plurality 5 of target miRNAs.
-78. (canceled)
A miRNA inhibitor comprising a nucleotide sequence of SEQ ID NO:
- 22, or SEQ ID NO:
- 24.
Dependencies are inferred from the claim text.