Bikash Pattnaik, PhD, Awarded NIH/New Investigator R01 Grant

Bikash Pattnaik, PhD, was recently awarded a 4-year New Investigator R01 award through the National Eye Institute (NEI) Audacious Goals Initiative from the National Institutes of Health/NEI for his project entitled, "Molecular Therapies for Lebers Congenital Amaurosis Caused by KCNJ13 Mutations." This award, in the amount of over $1.5 million, will allow investigations into causes of Leber's Congenital Amaurosis (LCA), an inherited retinal degenerative disease characterized by severe loss of vision at birth. Investigators will use gene editing of human iPSC-RPE (retina pigment epithelium) cells in the LCA16 drug discovery pathway and bypass the traditional use of animal disease models before clinical trials are attempted. The goal is to generate novel therapeutic strategies to improve the clinical outcomes of ion channelopathies.