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A Structure Based Approach For Defining Ligand Interaction Requirements With Atp-Binding Cassette Proteins, Christina A. Le
A Structure Based Approach For Defining Ligand Interaction Requirements With Atp-Binding Cassette Proteins, Christina A. Le
All ETDs from UAB
Membrane proteins belonging to the ATP-Binding Cassette (ABC) superfamily are responsible for transporting substrates into or out of cells. ABC transporters, such as P-glycoprotein (Pgp/ABCB1) defend cells by the active efflux of many chemically diverse cytotoxic compounds. The ABC protein, CFTR/ABCC7, is a passive chloride ion channel that provides fluid homeostasis. Pgp and CFTR of are high clinical interest since mutations in CFTR can result in Cystic Fibrosis (CF) and upregulation of Pgp in tumor cells can result in multidrug resistance (MDR). There has been a major effort to develop clinical drugs to treat CF by either repairing defects in …
Novel Approaches To Enhance Translational Readthrough Efficacy For Cystic Fibrosis Nonsense Mutations, Jyoti Sharma
Novel Approaches To Enhance Translational Readthrough Efficacy For Cystic Fibrosis Nonsense Mutations, Jyoti Sharma
All ETDs from UAB
Cystic fibrosis (CF) is a monogenic autosomal recessive disease caused by over 1,900 naturally occurring variants in the CF transmembrane conductance regulator (CFTR). CFTR is an epithelial anion channel which regulates the movement of chloride and bicarbonate ions. Mutations in the CFTR causes diminished CFTR protein and/or reduced CFTR function that lead to clinical manifestations. These include severe epithelial dysfunction of multiple tissues, including the lungs, intestine, pancreas, and reproductive organs. Premature termination codons (PTC), or nonsense mutations, are among the most severe CFTR variants and occur in ~11% of the CF population. PTCs are caused by the presence of …