Recombinant aav vectors useful for reducing immunity against transgene products
AssigneeUniversity of Massachusetts
InventorsGuangping Gao, Phillip D. Zamore, Shaoyong Li
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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.
Claims63
A method of delivering a transgene to target cells of a subject, the method comprising administering to the subject a recombinant Adeno-Associated Virus (rAAV) comprising a transgene engineered to express an RNA transcript that comprises a binding site of at least one immune-associated miRNA, wherein the rAAV infects target cells of the subject thereby delivering the transgene to the target cells.
A recombinant Adeno-Associated Virus (rAAV) comprising a transgene encoding an RNA transcript that comprises binding sites for at least two immune-associated miRNAs.
A nucleic acid comprising transgene engineered to express an RNA transcript that comprises binding sites for at least two immune-associated miRNAs, wherein the transgene is flanked by inverted terminal repeat sequences of an adeno-associated virus.
A method of delivering a transgene to target cells of a subject, the method comprising administering to the subject a recombinant Adeno-Associated Virus (rAAV) comprising a transgene engineered to express a recombinant protein that comprises Glycine-Alanine repeats.
Dependencies are inferred from the claim text.