Patent ID: 11920133
Assignee: UNIVERSITY OF MASSACHUSETTS
Field: Pharmaceuticals (Chemistry)
Classification: CPC C  A | IPC A  C

Claim 0:
1. A method of treating AAT-deficiency in a subject in need thereof, comprising administering to the subject an effective amount of an isolated nucleic acid, wherein the isolated nucleic acid comprises:
a) a first region that encodes one or more first miRNAs comprising a nucleic acid having sufficient sequence complementarity 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 complementarity to hybridize with and inhibit expression of the exogenous mRNA;
wherein the first region is positioned within an untranslated portion of the second region;
wherein the first protein is an AAT protein; and
wherein the second protein is a hardened AAT protein.