Development of Therapeutic Strategies To Combat Serpinopathies
Several therapeutic approaches are in use or under investigation to treat the most common serpinopathy; antitrypsin deficiency. Antitrypsin augmentation therapy is approved for severe antitrypsin deficiency-related pulmonary emphysema. Lung and / or liver transplantation is also used to treat severe antitrypsin deficiency-related disease. In animal models, gene targeting in induced pluripotent stem cells has successfully been deployed to correct the Z-antitrypsin defect and to restore the ability of the mammalian liver to secrete active antitrypsin. A number of groups have also reported small molecules that block antitrypsin polymerisation in vitro.
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