[From the laboratory to the clinic: CFTR and the therapeutic options for cystic fibrosis].

Article

[From the laboratory to the clinic: CFTR and the therapeutic options for cystic fibrosis].

Anales de medicina interna (Madrid, Spain : 1984) - 1 Feb 2000

Mayol J, Alvarez de Arcaya Vicente A, Arbeo Escolar A M, Peña Soria M J, Alvarez Fernández-Represa J

Abstract excerpt

Cystic fibrosis is a common lethal heritable disorder, caused by a defect in a chloride channel protein, namely CFTR. After the identification of the gene and its product by positional cloning (on chromosome 7), CFTR has been characterized as a low conductance (8-10 pSiemens) linear chloride channel, which appears to regulate other apical transport proteins. Two therapeutic options are reviewed: gene transfection...

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