Preclinical evaluation demonstrates reduction of Z alpha-1 antitrypsin polymers in human hepatocytes and mice using LRRK2 inhibitors, suggesting a targeted strategy for protein misfolding.
Alpha-1 antitrypsin deficiency (A1ATD) is a life-threatening condition caused by the inheritance of the serpin family A member 1 "Z" genetic variant driving alpha-1 antitrypsin (AAT) protein misfolding in hepatocytes. There are no approved medicines for this disease.
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Kent et al. (2024) studied this question.
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