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Article

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

7 Nov 2018

Abstract excerpt

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...

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PMID: 30425265
DOI: 10.1038/s41598-018-34960-0

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