Chemically modified hCFTR mRNAs recuperate lung function in a mouse model of cystic fibrosis
Chemically modified hCFTR mRNAs recuperate lung function in a mouse model of cystic fibrosis
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Author / Creator
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 and Kormann, Michael S. D.
Publisher
London: Nature Publishing Group UK
Journal title
Language
English
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Publication information
Publisher
London: Nature Publishing Group UK
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Scope and Contents
Contents
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 explor...
Alternative Titles
Full title
Chemically modified hCFTR mRNAs recuperate lung function in a mouse model of cystic fibrosis
Authors, Artists and Contributors
Author / Creator
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.
Identifiers
Primary Identifiers
Record Identifier
TN_cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_6233194
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_6233194
Other Identifiers
ISSN
2045-2322
E-ISSN
2045-2322
DOI
10.1038/s41598-018-34960-0