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Chemically modified hCFTR mRNAs recuperate lung function in a mouse model of cystic fibrosis.
Haque, A K M Ashiqul Dewerth, Alexander Antony, Justin S Riethmüller, Joachim Schweizer, Georg R Weinmann, Petra Latifi, Ngadhnjim Yasar, Hanzey Pedemonte, Nicoletta Sondo, Elvira
Haque, A K M Ashiqul
Dewerth, Alexander
Antony, Justin S
Riethmüller, Joachim
Schweizer, Georg R
Weinmann, Petra
Latifi, Ngadhnjim
Yasar, Hanzey
Pedemonte, Nicoletta
Sondo, Elvira
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2018-11-13
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Gene therapy has always been a promising therapeutic approach for Cystic Fibrosis (CF). However, numerous trials using DNA or viral vectors encoding the correct protein resulted in a general low efficacy. In the last years, chemically modified messenger RNA (cmRNA) has been proven to be a highly potent, pulmonary drug. Consequently, we first explored the expression, function and immunogenicity of human (h)CFTR encoded by cmRNA
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2045-2322
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Attribution-NonCommercial-ShareAlike 4.0 International