Cystic Fibrosis: Alternative Approaches to the Treatment of a Genetic Disease

D. J. Porteous, J. R. Dorin, B. J. Wainwright, S. Webb, S. J. Delaney, S. N. Smith, E. Farini, E. W. F. W. Alton, R. Farley, A demonstration using mouse models that successful gene therapy for cystic fibrosis requires only partial gene correction Gene Therapy. ,vol. 3, pp. 797- 801 ,(1996)

Marcus A. Mall, Role of the amiloride-sensitive epithelial Na+ channel in the pathogenesis and as a therapeutic target for cystic fibrosis lung disease. Experimental Physiology. ,vol. 94, pp. 171- 174 ,(2009) , 10.1113/EXPPHYSIOL.2008.042994

Tzyh-Chang Hwang, David N. Sheppard, Gating of the CFTR Cl− channel by ATP-driven nucleotide-binding domain dimerisation The Journal of Physiology. ,vol. 587, pp. 2151- 2161 ,(2009) , 10.1113/JPHYSIOL.2009.171595

Gerald B Pier, Martha Grout, Tanweer Zaidi, Gloria Meluleni, Simone S Mueschenborn, George Banting, Rosemary Ratcliff, Martin J Evans, William H Colledge, None, Salmonella typhi uses CFTR to enter intestinal epithelial cells. Nature. ,vol. 393, pp. 79- 82 ,(1998) , 10.1038/30006

DAVID N. SHEPPARD, MICHAEL J. WELSH, Structure and Function of the CFTR Chloride Channel Physiological Reviews. ,vol. 79, pp. 23- 45 ,(1999) , 10.1152/PHYSREV.1999.79.1.S23

ERIK M. SCHWIEBERT, DALE J. BENOS, MARIE E. EGAN, M. JACKSON STUTTS, WILLIAM B. GUGGINO, CFTR Is a Conductance Regulator as well as a Chloride Channel Physiological Reviews. ,vol. 79, pp. 145- 166 ,(1999) , 10.1152/PHYSREV.1999.79.1.S145

S. Gabriel, K. Brigman, B. Koller, R. Boucher, M. Stutts, Cystic fibrosis heterozygote resistance to cholera toxin in the cystic fibrosis mouse model Science. ,vol. 266, pp. 107- 109 ,(1994) , 10.1126/SCIENCE.7524148

Johanna M Rommens, Michael C Iannuzzi, Bat-sheva Kerem, Mitchell L Drumm, Georg Melmer, Michael Dean, Richard Rozmahel, Jeffery L Cole, Dara Kennedy, Noriko Hidaka, Martha Zsiga, Manuel Buchwald, Lap-Chee Tsui, John R Riordan, Francis S Collins, Identification of the cystic fibrosis gene: chromosome walking and jumping Science. ,vol. 245, pp. 1059- 1065 ,(1989) , 10.1126/SCIENCE.2772657

Bob Lubamba, Hugues Lecourt, Jean Lebacq, Patrick Lebecque, Hugo De Jonge, Pierre Wallemacq, Teresinha Leal, Preclinical evidence that sildenafil and vardenafil activate chloride transport in cystic fibrosis American Journal of Respiratory and Critical Care Medicine. ,vol. 177, pp. 506- 515 ,(2008) , 10.1164/RCCM.200703-344OC

Margaret A. McPherson, Malcolm M.C. Pereira, Chris Lloyd Mills, Kenneth J. Murray, Robert L. Dormer, A cyclic nucleotide PDE5 inhibitor corrects defective mucin secretion in submandibular cells containing antibody directed against the cystic fibrosis transmembrane conductance regulator protein FEBS Letters. ,vol. 464, pp. 48- 52 ,(1999) , 10.1016/S0014-5793(99)01672-5