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- W4386284927 endingPage "1835" @default.
- W4386284927 startingPage "1835" @default.
- W4386284927 abstract "Cystic fibrosis is a genetic disease that results in progressive multi-organ manifestations with predominance in the respiratory and gastrointestinal systems. The significant morbidity and mortality seen in the CF population has been the driving force urging the CF research community to further advance treatments to slow disease progression and, in turn, prolong life expectancy. Enormous strides in medical advancements have translated to improvement in quality of life, symptom burden, and survival; however, there is still no cure. This review discusses the most current mainstay treatments and anticipated therapeutics in the CF drug development pipeline within the mechanisms of mucociliary clearance, anti-inflammatory and anti-infective therapies, restoration of the cystic fibrosis transmembrane conductance regulator (CFTR) protein (also known as highly effective modulator therapy (HEMT)), and genetic therapies. Ribonucleic acid (RNA) therapy, gene transfer, and gene editing are being explored in the hopes of developing a treatment and potential cure for people with CF, particularly for those not responsive to HEMT." @default.
- W4386284927 created "2023-08-31" @default.
- W4386284927 creator A5017830229 @default.
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- W4386284927 creator A5076319792 @default.
- W4386284927 creator A5077484907 @default.
- W4386284927 creator A5088101789 @default.
- W4386284927 date "2023-08-30" @default.
- W4386284927 modified "2023-10-17" @default.
- W4386284927 title "Advances in the Cystic Fibrosis Drug Development Pipeline" @default.
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- W4386284927 doi "https://doi.org/10.3390/life13091835" @default.
- W4386284927 hasPubMedId "https://pubmed.ncbi.nlm.nih.gov/37763239" @default.
- W4386284927 hasPublicationYear "2023" @default.
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