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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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Detalles Bibliográficos
Autores principales: 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.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2018
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6233194/
https://www.ncbi.nlm.nih.gov/pubmed/30425265
http://dx.doi.org/10.1038/s41598-018-34960-0