Patient personalized translational tools in cystic fibrosis to transform data from bench to bed-side and back
Kavisha Arora, Fanmuyi Yang, John J. Brewington, Gary L. McPhail, Alexander R. Cortez, Nambirajan Sundaram, Yashaswini Ramananda, Herbert Luke Ogden, Michael A. Helmrath, John P. Clancy, Anjaparavanda P. Naren
2021American Journal of Physiology-Gastrointestinal and Liver Physiology11 citationsDOIOpen Access PDF
Abstract
In this study, we report an example of laboratory models informing clinical care for rare CF mutation patient, with subsequent recapitulation of clinical benefit in a unique and disease relevant, human-derived in vivo model, effectively translating data from the lab to the clinic and back. This extensive work outlines a potential platform to identify patient-specific therapies and to understand relevant developmental abnormalities associated with CF disease.
Topics & Concepts
IvacaftorCystic fibrosisCystic fibrosis transmembrane conductance regulatorMedicineMutationGenetic testingPhenotypeClinical trialIn vivoBioinformaticsInternal medicineBiologyGeneGeneticsCystic Fibrosis Research AdvancesNeonatal Respiratory Health ResearchChild Nutrition and Feeding Issues