Sod1 dual expression vectors and uses thereof
AssigneeUniversity of Massachusetts
InventorsChristian Mueller, Robert H. Brown, JR.
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Abstract
In some aspects, the disclosure relates to compositions and methods useful for inhibiting SOD1 expression in cells (e.g., cells of a subject). In some embodiments, the disclosure describes isolated nucleic acids engineered to express an inhibitory nucleic acid targeting endogenous SOD1 and an mRNA encoding a hardened SOD1 protein. In some embodiments, compositions and methods described by the disclosure are useful for treating Amyotrophic Lateral Sclerosis (ALS) in a subject.
Claims27
1-30. (canceled)
A method for inhibiting SOD1 expression in a cell, the method comprising delivering to a cell an isolated nucleic acid comprising:
- 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 SOD1 protein;
- and
- a second region encoding an exogenous mRNA that encodes a wild-type SOD1 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 wild-type SOD1 protein is encoded by a sequence comprising the sequence set forth in SEQ ID NO:
- 7.
A method for treating a subject having or suspected of having amyotrophic lateral sclerosis (ALS), the method comprising administering to the subject an effective amount of an isolated nucleic acid comprising:
- a first region that encodes one or more first miRNAs comprising a nucleic acid having sufficient sequence complementary with an endogenous mRNA of the subject to hybridize with and inhibit expression of the endogenous mRNA, wherein the endogenous mRNA encodes a SOD1 protein;
- and
- a second region encoding an exogenous mRNA that encodes a wild-type SOD1 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 wild-type SOD1 protein is encoded by a sequence comprising the sequence set forth in SEQ ID NO:
- 7.
Dependencies are inferred from the claim text.