Bikash Pattnaik, PhD, and UW Investigators Awarded R24 Grant from NIH

Bikash Pattnaik, PhD
Bikash Pattnaik, PhD

Bikash Pattnaik, PhD, and Co-Principal Investigators, Shaoqin “Sarah” Gong, PhD, Biomedical Engineering and Wisconsin Institute for Discovery, David Gamm, MD, PhD, Department of Visual Sciences and Ophthalmology, and Christopher Ahern, PhD, University of Iowa, recently received a 5-year, $7.7 million R24 award from the National Institutes of Health/National Eye Institute (NIH/NEI) as part of the NEI Translational Research Program to Develop Novel Therapies and Devices for the Treatment of Visual System Disorders. Their project, “Restoring Vision with High-Fidelity Nonsense Codon Correction,” will develop a nucleic acid-based therapy using anticodon edited transfer RNA (ace-tRNA) to target nonsense mutations that cause blindness. A nonsense mutation is a point mutation in the DNA sequence that deleteriously stops healthy protein synthesis prematurely and the disease phenotype is often severe. Currently, there are no FDA-approved treatments and only a limited number of therapies for nonsense mutations being tested in human clinical trials. This novel project aims to advance ace-tRNA technology toward clinical trials for a wide range of genetic diseases that cause blindness.