Non-viral mRNA cystic fibrosis therapies and their ongoing clinical trials.

Abstract excerpt

INTRODUCTION: Cystic fibrosis (CF) is a genetic disease caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, leading to impaired ion transport by the CFTR protein and accumulation of thick, sticky mucus, resulting in chronic lung disease and other organ complications. Messenger ribonucleic acid (mRNA) therapies hold great potential for CF, as they can be used for both CFTR...

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