Article

Modified h <i>CFTR</i> mRNA restores normal lung function in a mouse model of cystic fibrosis

2017-10-13

Abstract excerpt

Being a classic monogenic disease, 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 effective drug. We thus explored the expression of human (h)CFTR en...

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Identifiers and source

Literature Corpus work: db658e80-598a-5469-96b7-bf5ac0f83630
DOI: 10.1101/202853

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