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    An Introduction to Cystic Fibrosis

    Landmark research characterizing genetic drivers and protein malformations underlying cystic fibrosis (CF) is successfully translating into new and effective targeted therapies. Recently approved agents that ‘pharmacologically repair’ defects in the cystic fibrosis transmembrane conductance regulator (CFTR) arising from the F508del-mutation have revolutionized the treatment paradigm in CF. Ongoing studies are exploring restorative interventions including gene-editing, messenger ribonucleic acid (mRNA), DNA and gene therapies. More effective strategies to manage infections and pulmonary exacerbations in CF remain an active area of clinical research.

    Browse our selection of video highlights and short articles from the conference hub, providing insights into the latest updates from major conferences and a selection of peer-reviewed articles from the journal portfolio.

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