KIR 7.1 gene therapy vectors and methods of using the same
AssigneeWisconsin Alumni Research Foundation
InventorsBikash Pattnaik, Pawan Shahi
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Abstract
The present invention is directed to gene therapy constructs and pharmaceutical compositions for the expression of Kir7.1. The gene therapy constructs include a vector comprising a promoter operably connected to a polynucleotide encoding a Kir7.1 polypeptide. Methods of treating a subject having a condition associated with insufficient expression or function of a Kir7.1 polypeptide are also provided.
Claims16
A gene therapy vector comprising a VMD2 promoter operably connected to a polynucleotide encoding a Kir7.1 polypeptide, wherein the VMD2 promoter comprises SEQ ID NO:
- 4 and is active in the retinal pigment epithelium (RPE) in the eye of a subject, and wherein the Kir7.1 polypeptide comprises SEQ ID NO:
- 1.
A lentiviral vector or adeno-associated viral (AAV) vector comprising SEQ ID NO:
- 6 (VMD2-Kir7.1).
Dependencies are inferred from the claim text.