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US 12,630,824 B2Granted
RAAV-based compositions and methods
AssigneeUniversity of Massachusetts
InventorsTerence Flotte, Christian Mueller, Phillip D. Zamore
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Abstract
The invention relates to isolated nucleic acids and rAAV-based compositions, methods and kits useful for treating genetic diseases (e.g., alpha-1 antitrypsin deficiency).
Claims20
A recombinant Adeno-Associated Virus (AAV) comprising an isolated nucleic acid comprising:
- (a) a first region that encodes one or more first miRNAs comprising a nucleic acid having sufficient sequence complementary with an endogenous mRNA of a subject to hybridize with and inhibit expression of the endogenous mRNA, wherein the endogenous mRNA encodes a first protein;
- and
- (b) a second region encoding an exogenous mRNA that encodes a second protein, wherein the second protein has an amino acid sequence that is at least 85% identical to the first protein,
- wherein the one or more first miRNAs do not comprise a nucleic acid having sufficient sequence complementary to hybridize with and inhibit expression of the exogenous mRNA, and wherein the first region is positioned within an untranslated portion of the second region.
1 more element
Dependencies are inferred from the claim text.