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

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^hCFTR in vitro and ex vivo, quantified the expression by flow cytometry, determined its function using a YFP based assay and checked the immune response in human whole blood. Similarly, we examined the function of cmRNA^hCFTR in vivo after intratracheal (i.t.) or intravenous (i.v.) injection of the assembled cmRNA^hCFTR together with Chi

SUBMITTER: Haque AKMA 

PROVIDER: S-EPMC6233194 | biostudies-literature | 2018 Nov

REPOSITORIES: biostudies-literature