Nationwide Children's Hospital
My name is Sriram Vaidyanathan. I am an assistant professor in the Department of Pediatrics at The Ohio State University and a principal investigator in the Center for Gene Therapy at the Abigail Wexner Research Institute. During my postdoctoral trianing in the lab of Dr. Matthew Porteus at Stanford University, I started working on the development of an autologous gene corrected airway stem cell therapy for CF. We first developed the methods to genome edit airway basal stem cells using CRISPR/Cas9 with high editing efficiencies and demonstrated the correction of the F508del mutation using this approach. In subsequent studies, I developed the methods to insert the full-length CFTR cDNA in the endogenous CFTR locus of airway basal stem cells. Thus mutation agnostic correction strategy is applicable to >99.5% of CF patients including those with mutations that are not responsive to modulators. We are working on completing preclinical studies needed to begin clinical trials testing the safety of this approach for treating CF sinus disease. As a new investigator, I will continue working on novel genome editing technologies and approaches to transplant gene corrected airway stem cells into the lower airways for treating CF lung disease.
Saturday, November 5, 2022
9:45 AM – 11:45 AM ET