Recombinant aav vectors useful for reducing immunity against transgene products
AssigneeUniversity of Massachusetts
InventorsGuangping Gao, Phillip D. Zamore, Shaoyong Li
Life of this patent
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Abstract
The disclosure in some aspects, relates to nucleic acids, compositions and kits useful for gene therapy with reduced immune response to transgene products.
Claims30
A method of delivering a transgene to a subject, the method comprising administering to the subject a recombinant Adeno-Associated Virus (rAAV) comprising the transgene, wherein the transgene comprises an RNA transcript that comprises two different immune-associated miRNA binding sites selected from the group consisting of:
- miR-15a, miR-16-1, miR-17, miR-18a, miR-19a, miR-20a, miR-19b-1, miR-21, miR-29a, miR-29b, miR-29c, miR-30b, miR-31, miR-34a, miR-92a-1, miR-106a, miR-125a, miR-125b, miR-126, miR-142-3p, miR-146a, miR-150, miR-155, miR-181a, miR-223 and miR-424.
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A recombinant Adeno-Associated Virus (rAAV) comprising a transgene encoding an RNA transcript that comprises binding sites for at least two different immune-associated miRNAs, wherein the at least two different immune-associated miRNA binding sites selected from the group consisting of:
- miR-15a, miR-16-1, miR-17, miR-18a, miR-19a, miR-20a, miR-19b-1, miR-21, miR-29a, miR-29b, miR-29c, miR-30b, miR-31, miR-34a, miR-92a-1, miR-106a, miR-125a, miR-125b, miR-126, miR-142-3p, miR-146a, miR-150, miR-155, miR-181a, miR-223 and miR-424.
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A nucleic acid comprising transgene engineered to express an RNA transcript that comprises binding sites for at least two different immune-associated miRNAs, wherein the transgene is flanked by inverted terminal repeat sequences of an adeno-associated virus, and wherein the at least two different immune-associated miRNA binding sites selected from the group consisting of:
- miR-15a, miR-16-1, miR-17, miR-18a, miR-19a, miR-20a, miR-19b-1, miR-21, miR-29a, miR-29b, miR-29c, miR-30b, miR-31, miR-34a, miR-92a-1, miR-106a, miR-125a, miR-125b, miR-126, miR-142-3p, miR-146a, miR-150, miR-155, miR-181a, miR-223 and miR-424.
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Dependencies are inferred from the claim text.