Article
Partial correction of endogenous DeltaF508 CFTR in human cystic fibrosis airway epithelia by spliceosome-mediated RNA trans-splicing.
Nature biotechnology - 1 Jan 2002
Liu Xiaoming, Jiang Qinshi, Mansfield S Gary, Puttaraju M, Zhang Yulong, Zhou Weihong, Cohn Jonathan A, Garcia-Blanco Mariano A, Mitchell Lloyd G, Engelhardt John F
Abstract excerpt
Spliceosome-mediated RNA trans-splicing (SMaRT) was investigated as a means for functionally correcting endogenous DeltaF508 cystic fibrosis transmembrane conductance regulator (CFTR) transcripts using in vitro human cystic fibrosis (CF) polarized airway epithelia and in vivo human CF bronchial xenografts. Recombinant adenovirus (Ad.CFTR-PTM) encoding a pre-therapeutic molecule (PTM) targeted to CFTR intron 9...
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