The National Heart, Lung, and Blood Institute (NHLBI) awarded a $629,319 Project Grant (CFDA 93.837 - Cardiovascular Diseases Research) to Emory University to investigate new ways to overcome treatment-resistant genetic defects in cystic fibrosis (CF). The research aims to identify cellular targets that can ameliorate CF by correcting the basic defects caused by premature termination codons in the CFTR gene. Specifically, the project will ascertain the effects of manipulating ribosomal proteins on the biogenesis of CFTR variants with premature stop codons, define the mechanisms by which this alters translation kinetics and mRNA surveillance, and determine the outcomes of ribosomal protein disruption in humanized CF mouse models. This multidisciplinary research is intended to establish translational control as a novel checkpoint during CFTR premature termination codon processing and identify specific ribosomal proteins that mediate this pathway, which could lead to new therapeutic approaches for CF and other genetic disorders.
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