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Du, M., Liu, X., Welch, E.M., Hirawat, S., Peltz, S.W. and Bedwell, D.M. (2008) PTC124 is an orally bioavailable compound that promotes suppression of the human CFTR-G542X nonsense allele in a CF mouse model. Proceedings of the National Academy of Sciences of the United States of America, 105, 2064-2069.
http://dx.doi.org/10.1073/pnas.0711795105
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