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A new era in the treatment of cystic fibrosis: correction of the underlying CFTR defect

Patients with strong behavioral commitment and clear milestone targets (such as reaching a specific weight before a major life event) often benefit most from defined treatment windows

A major limiting factor in the development of new treatments is the lack of predictive pre-clinical models that accurately reflect human disease with regard to liver histology, pathophysiology, and metabolic abnormalities

This is unsurprising as the RPE has a high level of metabolic activity, and therefore oxygen demand, making it a significant source of ROS [17]