Isolation of novel AAV'S and uses thereof
AssigneeUniversity of Massachusetts
InventorsGuangping Gao, Phillip D. Zamore
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Abstract
The invention in some aspects relates to isolated nucleic acids, compositions, and kits useful for identifying adeno-associated viruses in cells. In some aspects, the invention provides kits and methods for producing somatic transgenic animal models using recombinant AAV (rAAV) to an animal having at least one transgene that expresses a small interfering nucleic acid or at least one binding site for a miRNA.
Claims14
A kit for producing a recombinant Adeno-Associated Virus (rAAV) that generates a somatic transgenic animal that expresses a transgene in a target tissue, the kit comprising:
- at least one container housing a recombinant AAV vector, wherein the recombinant AAV vector comprises a transgene that expresses an RNA transcript that comprises at least one binding site for an miRNA that is expressed in cells of an off-target tissue of the animal, wherein, when the miRNA and the RNA transcript are present together in cells of the off-target tissue, the miRNA hybridizes to the at least one binding site of the RNA transcript and inhibits activigty of the RNA transcript wherein the miRNA is miR-122 and the off-target tissue is liver;
- at least one container housing a rAAV packaging component, wherein the packing componenet comprisies a host cell comprising at least one cap gene encoding a capsid protein of AAV9;
- and
- instructions for constructing and packaging the rAAV, wherein the rAAV transduces cells of the target tissue.
A recombinant adeno-associated virus (rAAV) comprising:
- an AAV9 in which is packaged a transgene that encodes an RNA transcript that comprises at least one binding site for miR-122 that inhibits expression of the RNA transcript in liver.
Dependencies are inferred from the claim text.