Isolation of novel AAV's and uses thereof
AssigneeUniversity of Massachusetts
InventorsGuangping Gao, Phillip D. Zamore
Life of this patent
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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.
Claims20
An isolated nucleic acid engineered to express an mRNA in a target tissue in a subject, wherein the mRNA contains a 3′ UTR comprising at least one binding site for a miRNA expressed endogenously in an off-target tissue in the subject, and wherein the mRNA is flanked by adeno-associated virus (AAV) inverted terminal repeat sequences (ITRs), wherein the nucleic acid encoding the mRNA is operably linked to a tissue-specific promoter, and wherein transduction of the target tissue with the isolated nucleic acid results in expression of the mRNA in the target tissue for at least three weeks.
A recombinant virus comprising a capsid housing an isolated nucleic acid engineered to express an mRNA in a target tissue in a subject, wherein the mRNA contains a 3′ UTR comprising at least one binding site for a miRNA expressed endogenously in an off-target tissue in the subject, and wherein the mRNA is flanked by adeno-associated virus (AAV) inverted terminal repeat sequences (ITRs), wherein the nucleic acid encoding the mRNA is operably linked to a tissue-specific promoter, and wherein transduction of the target tissue with the isolated nucleic acid results in expression of the mRNA in the target tissue for at least three weeks.
A syringe containing an injectable aqueous solution that comprises an isolated nucleic acid engineered to express an mRNA in cells of a target tissue, wherein the mRNA contains a 3′ UTR comprising one or more binding sites for an miRNA, wherein the mRNA is flanked by adeno-associated virus (AAV) inverted terminal repeat sequences (ITRs), wherein the isolated nucleic acid is packaged in an AAV viral capsid, wherein the nucleic acid expressing the mRNA is operably linked to a tissue-specific promoter, and wherein transduction of the target tissue with the isolated nucleic acid results in expression of the mRNA in the target tissue for at least three weeks.
A method of delivering a transgene to a subject having a tumor, the method comprising administering by injection into the tumor an aqueous solution that comprises an isolated nucleic acid containing a transgene engineered to express an mRNA in cells of the tumor, wherein the mRNA contains a 3′ UTR comprising one or more binding sites for an miRNA, wherein the transgene is flanked by adeno-associated virus (AAV) inverted terminal repeat sequences (ITRs), and wherein the isolated nucleic acid is packaged in an AAV viral capsid wherein the nucleic acid that expresses the mRNA is operably linked to a tissue-specific promoter, and wherein injection of the aqueous solution with the isolated nucleic acid results in expression of the mRNA in the tumor for at least three weeks.
Dependencies are inferred from the claim text.