Affiliation: University of Toronto

My laboratory has published >160 papers including those focusing on the molecular basis for disease caused by Cystic Fibrosis and the mechanism underlying therapeutic rescue by small molecule modulators. My lab has also defined putative mechanisms underlying modification of Cystic Fibrosis disease severity by genetic modifiers-namely, the amino acid transporter, SLC6A14.  We have been instrumental in developing methods for CF disease and therapy modeling using stem cell derived tissues through the creation of novel phenotypic tools for interrogating ion channel function for these tissues. Most recently, in order to enable genome/transcriptome/phenome wide interrogation of patient-specific barriers to therapeutic rescue, I initiated funding for and now, co-direct the unique bioresource, called the CFIT (Cystic Fibrosis Program for Individualized Therapy), 100 cell line project. This bioresource is comprised of airway samples and paired iPSC lines from more than 100 donors (with or without pre-existing disease) with links to whole genome sequence and clinical data. Our program enables CF therapy discovery by academic and industry groups as well as the training of highly qualified personnel.