Gentamicin-induced correction of CFTR function in patients with cystic fibrosis and CFTR stop mutations.

  title={Gentamicin-induced correction of CFTR function in patients with cystic fibrosis and CFTR stop mutations.},
  author={Michael Wilschanski and Yaacov Yahav and Yasmin Yaacov and Hannah Blau and Lea Bentur and Joseph Rivlin and Micha Aviram and Tali Bdolah-Abram and Zsuzsa Bebők and Liat Shushi and Batsheva Kerem and Eitan Kerem},
  journal={The New England journal of medicine},
  volume={349 15},
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 nasal epithelium of patients with cystic fibrosis could result in the expression of… CONTINUE READING
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