Chemically modified hCFTR mRNAs recuperate lung function in a mouse model of cystic fibrosis.
dc.contributor.author | Haque, A K M Ashiqul | |
dc.contributor.author | Dewerth, Alexander | |
dc.contributor.author | Antony, Justin S | |
dc.contributor.author | Riethmüller, Joachim | |
dc.contributor.author | Schweizer, Georg R | |
dc.contributor.author | Weinmann, Petra | |
dc.contributor.author | Latifi, Ngadhnjim | |
dc.contributor.author | Yasar, Hanzey | |
dc.contributor.author | Pedemonte, Nicoletta | |
dc.contributor.author | Sondo, Elvira | |
dc.contributor.author | Weidensee, Brian | |
dc.contributor.author | Ralhan, Anjali | |
dc.contributor.author | Laval, Julie | |
dc.contributor.author | Schlegel, Patrick | |
dc.contributor.author | Seitz, Christian | |
dc.contributor.author | Loretz, Brigitta | |
dc.contributor.author | Lehr, Claus-Michael | |
dc.contributor.author | Handgretinger, Rupert | |
dc.contributor.author | Kormann, Michael S D | |
dc.date.accessioned | 2018-12-19T14:51:50Z | |
dc.date.available | 2018-12-19T14:51:50Z | |
dc.date.issued | 2018-11-13 | |
dc.identifier.issn | 2045-2322 | |
dc.identifier.pmid | 30425265 | |
dc.identifier.doi | 10.1038/s41598-018-34960-0 | |
dc.identifier.uri | http://hdl.handle.net/10033/621624 | |
dc.description.abstract | 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 | en_US |
dc.publisher | Nature publishing group | en_US |
dc.rights | Attribution-NonCommercial-ShareAlike 4.0 International | * |
dc.rights.uri | http://creativecommons.org/licenses/by-nc-sa/4.0/ | * |
dc.title | Chemically modified hCFTR mRNAs recuperate lung function in a mouse model of cystic fibrosis. | en_US |
dc.type | Article | en_US |
dc.contributor.department | HIPS, Helmholtz-Institut füt Pharmazeutische Forschung Saarland, Universitätscampus E8.1 66123 Saarbrücken, Germany. | en_US |
refterms.dateFOA | 2018-12-19T14:51:50Z | |
dc.source.journaltitle | Scientific reports |