Therapeutics & Vaccines
Kir 7.1 Gene Therapy Vectors And Methods Of Using The Same
WARF: P180340US05
Inventors: Bikash Pattnaik, Pawan Shahi
The Invention
The present invention from UW Madison researchers is directed to improved AAV gene therapy constructs and pharmaceutical compositions for the expression of Kir7.1. The gene therapy constructs are particularly AAV vector comprising a promoter operably connected to a polynucleotide encoding a Kir7.1 polypeptide which is capable of being expressed in retinal pigment epithelium cells. Methods of treating a subject having a condition associated with insufficient expression or function of a Kir7.1 polypeptide are also provided.