Article
Gentamicin-induced correction of CFTR function in patients with cystic fibrosis and CFTR stop mutations.
The New England journal of medicine - 9 Oct 2003
Wilschanski Michael, Yahav Yaacov, Yaacov Yasmin, Blau Hannah, Bentur Lea, Rivlin Joseph, Aviram Micha, Bdolah-Abram Tali, Bebok Zsuzsa, Shushi Liat, Kerem Batsheva, Kerem Eitan
Abstract excerpt
BACKGROUND: Mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene containing a premature termination signal cause a deficiency or absence of functional chloride-channel activity. Aminoglycoside antibiotics can suppress premature termination codons, thus permitting translation to continue to the normal end of the transcript. We assessed whether topical administration of gentamicin to the...
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