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Genome-wide association studies with large patient cohorts have identified genetic variants of specific loci associated with disease severity, called CF modifier genes, that contribute to phenotypic variability [see recent reviews (Lim et al., 2018

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GAGs are a group of polysaccharide molecules, also known as mucopolysaccharides, that create the foundational structure of the extracellular matrix (ECM), together with other components such as collagen, elastin, fibronectin, and laminin Biomechanically, the layer of GAGs functions as a hydrated gel that provides cushioning and resists compressive forces

They must also pass through the liver where metabolism often occurs before reaching the bloodstream, which reduces their effectiveness

R.ChenH.ChipotC.KohlmeyerA.et al (2024)

doi: 10.1111/jam.14551 90