CareLit · DOI-Nachweis · Pneumologie
Modeling Cystic Fibrosis in vitro: A new possible platform for patient customised drug screening and ex-vivo gene therapy?
DOI 10.1055/s-0034-1376824
Bibliografische Angaben
Nachweis in CareLit
Autor:innenR Haller, S Ulrich, S Weinreich, S Merkert, C Bednarski, C Mauritz, U Martin
ZeitschriftPneumologie
Ausgabe06/2014
Jahrgang / SeitenNicht angegeben
Kernaussage aus der Literatur
Abstract-Vorschau
Cystic fibrosis (CF) is the most common lethal monogenic recessive disease in the caucasian population. Over 1900 mutations are known in the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) gene, but the most common mutation is the F508del resulting in misfolding of the CFTR protein. Until today, classical gene therapy trials have not been successful in CF patients, and currently approved CFTR modulators a…
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Zitierempfehlung
R Haller, S Ulrich, S Weinreich, S Merkert, C Bednarski, C Mauritz, U Martin (2014) Modeling Cystic Fibrosis in vitro: A new possible platform for patient customised drug screening and ex-vivo gene therapy?. Pneumologie (06). https://doi.org/10.1055/s-0034-1376824
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