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Cystic fibrosis transmembrane conductance regulator intracellular processing, trafficking, and opportunities for mutation-specific treatment.
Rogan MP, Stoltz DA, Hornick DB. Rogan MP, et al. Chest. 2011 Jun;139(6):1480-1490. doi: 10.1378/chest.10-2077. Chest. 2011. PMID: 21652558 Review.
We review the results of encouraging clinical trials examining orally administered therapeutics, including agents that promote read-through of class I mutations (premature termination codons); correctors, which overcome the CFTR misfolding that characterizes the common class II m …
We review the results of encouraging clinical trials examining orally administered therapeutics, including agents that promote read-through …