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CFTR mRNA Based Gene Therapy for cystic fibrosis (CF)

A Yamamoto, M Illenyi, M Kormann, J Rosenecker, C Rudolph · Pneumologie · Ausgabe 01/2008

DOI 10.1055/s-2007-1032308

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Autor:innenA Yamamoto, M Illenyi, M Kormann, J Rosenecker, C Rudolph
ZeitschriftPneumologie
Ausgabe01/2008
Jahrgang / SeitenNicht angegeben

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Abstract-Vorschau

Cystic fibrosis (CF) is a lethal autosomal recessive genetic disease which affects over 70, 000 people in the world. The responsible gene for CF was identified in 1989, so called CFTR (cystic fibrosis transmembrane conductance -regulator), and CF is caused by over 1300 mutations in CFTR. Among these mutations, deletion phenylalanine 508 (dF508) causes more than 90% cases of CF. Main pathological features are thick a…

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A Yamamoto, M Illenyi, M Kormann, J Rosenecker, C Rudolph (2008) CFTR mRNA Based Gene Therapy for cystic fibrosis (CF). Pneumologie (01). https://doi.org/10.1055/s-2007-1032308

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