Codon-optimized reduced-size atp7a cdna and uses for treatment of copper transport disorders
AssigneeThe United States of America, as represented by the Secretary, Dept. of Health and Human Services
InventorStephen G. Kaler
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Abstract
Disclosed herein are codon-optimized nucleic acids encoding a reduced-size ATP7A protein. Also disclosed are vectors and recombinant viruses (such as recombinant adeno-associated viruses) including the codon-optimized nucleic acids encoding the reduced-size ATP7A protein and compositions including the disclosed vectors and viruses. Further disclosed herein are methods of treating copper transport disorders, for example by administering a disclosed nucleic acid, vector, or recombinant virus to a subject with a copper transport disorder, such as Menkes disease, occipital horn syndrome, or ATP7A-related distal motor neuropathy.
Claims20
A recombinant adeno-associated virus (rAAV) vector comprising a nucleic acid encoding an ATP7A protein, wherein the nucleic acid is operably linked to a promoter, and wherein the nucleic acid comprises SEQ ID NO:
- 1.
A composition comprising:
- a recombinant adeno-associated virus (rAAV) vector comprising a nucleic acid encoding an ATP7A protein, wherein the nucleic acid is operably linked to a promoter, and wherein the nucleic acid comprises SEQ ID NO:
- 1;
- and
- a pharmaceutically acceptable carrier.
A host cell comprising a recombinant adeno-associated virus (rAAV) vector comprising a nucleic acid encoding an ATP7A protein, wherein the nucleic acid is operably linked to a promoter, and wherein the nucleic acid comprises SEQ ID NO:
- 1, wherein the rAAV is within or on the host cell.
Dependencies are inferred from the claim text.