Modified aav constructs and uses thereof
AssigneeUniversity of Massachusetts
InventorsGuangping Gao, Jun Xie, Phillip D. Zamore
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Abstract
The present disclosure relates to the field of rAAV delivery of transgenes. In some aspects, the disclosure relates to RNAi. Provided herein are recombinant adeno-associated virus (rAAV) vectors comprising modified ITRs. In some embodiments, the modified ITRs comprise a sequence encoding a shRNA, miRNA, or AmiRNA.
Claims33
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An isolated nucleic acid having one inverted terminal repeat at a first terminus and a promoter operably linked with a sequence encoding a hairpin-forming RNA at a second terminus, wherein the isolated nucleic acid is configured for forming a self-complementary AAV (scAAV) vector.
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A preparation comprising a plurality of rAAVs, wherein at least 80% of the rAAVs comprise a non-truncated genome comprising a sequence encoding an artificial miRNA (AmiRNA).
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A self-complementary adeno-associated virus (scAAV) comprising:
- (i) a viral genome comprising a nucleic acid sequence encoding at least one inverted terminal repeat and a promoter operably linked with a nucleic acid sequence encoding a hairpin-forming RNA;
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- (ii) at least one AAV capsid protein serotype.
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Dependencies are inferred from the claim text.