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

Claim 10:
11. A method of treating a genetic disease associated with an AAT-deficiency in a subject in need thereof, comprising administering an effective amount of an isolated nucleic acid to a subject, wherein the isolated nucleic acid comprises:
a) a first region encoding 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 downstream of a portion of the second region encoding the poly-A tail of the exogenous mRNA;
wherein the subject having the AAT-deficiency produces mutant AAT protein or insufficient amounts of AAT protein;
wherein the first protein is an AAT protein; and
wherein the second protein is a hardened AAT protein.