NIH R01 · 2024
Modulation of Lung Disease by Genetic/Epigenetic Profiling
Project Summary/Abstract: Therapeutic management of lung disorders triggered by the loss-of-function of the cystic fibrosis (CF) transmembrane conductance regulator (CFTR) function in response to leading to CF are challenged by genetic and epigenetic diversity found in the CF population. The highly effective modulator therapy (HEMT) Trikafta has a pronounced but incomplete and variable impact on the pathology of disease in the clinic. We now need to discover new approaches to further improve clinical outcome. CF is not a simple monogenic disease but rather a complex disease impacted by membrane trafficking and channel function of CFTR- as well as diverse clinical features including…
From the public funding record at NIH RePORTER. Describes the funded project, not the reviews below.