Bikash Pattnaik and collaborators receive grant from Spotlight Therapeutics

Bikash Pattnaik, PhD
Bikash Pattnaik, PhD

Co-Principal Investigators, Bikash Pattnaik, PhD, associate professor, Neonatology and Newborn Nursery, and Krishanu Saha, PhD, Department of Biomedical Engineering, and Co-Investigator Pawan Shahi, PhD, assistant scientist,Neonatology and Newborn Nursery, received a six-month $76,352 award from Spotlight Therapeutics for their project, “Using TAGE to target gene mutations in mice retina.” The project will explore the delivery of Spotlight, Targeted Active Gene Editors (TAGE) in Leber congenital amaurosis (LCA16)-affected mice to target the KCNJ13 gene. The KCNJ13 gene codes for Kir7.1, an inwardly-rectifying potassium channel protein that is essential for vision. A mutated KCNJ13 gene is associated with a potassium channel defect in the retinal pigment epithelium, which causes LCA16. This approach provides direct, irreversible conversion to correct a genetic mutation of targeted genes and another potential therapy to treat LCA16 and other genetic disorders.