Chemically modified hCFTR mRNAs recuperate lung function in a mouse model of cystic fibrosis

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 pot...

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Détails bibliographiques
Publié dans:Sci Rep
Auteurs principaux: 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, Weidensee, Brian, Ralhan, Anjali, Laval, Julie, Schlegel, Patrick, Seitz, Christian, Loretz, Brigitta, Lehr, Claus-Michael, Handgretinger, Rupert, Kormann, Michael S. D.
Format: Artigo
Langue:Inglês
Publié: Nature Publishing Group UK 2018
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Article
Accès en ligne:https://ncbi.nlm.nih.gov/pmc/articles/PMC6233194/
https://ncbi.nlm.nih.gov/pubmed/30425265
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/s41598-018-34960-0
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