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; Weidensee, Brian; Ralhan, Anjali; Laval, Julie; Schlegel, Patrick; Seitz, Christian; Loretz, Brigitta; Lehr, Claus-Michael; Handgretinger, Rupert; Kormann, Michael S D

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Issue Date

2018-11-13

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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

Affiliation

HIPS, Helmholtz-Institut füt Pharmazeutische Forschung Saarland, Universitätscampus E8.1 66123 Saarbrücken, Germany.

Type

Article

ISSN

2045-2322

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publications of the research group drug delivery ([HIPS] DDEL)

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Except where otherwise noted, this item's license is described as Attribution-NonCommercial-ShareAlike 4.0 International