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Alpha1-antitrypsin Deficiency

Tundra lists 1 Alpha1-antitrypsin Deficiency clinical trial. Each listing includes eligibility criteria, study locations, and direct links to research sites in the Tundra directory.

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ENROLLING BY INVITATION

NCT06892236

Preparation of IPSC for Cell Gene Editing for the Treatment of AATD

Alpha 1-antitrypsin (AAT) deficiency is a genetic condition that leads to lung and/or liver diseases; current treatment of weekly augmentation of AAT addresses only lung diseases with moderate efficacy. Novel treatments based on gene editing can restore physiological levels of AAT and address lung and liver disease. The aim is to generate induced pluripotent stem cells (iPSC) from blood and urine of patients with different severe Alpha1-antitrypsin deficiency (AATD) genotypes. Further, the iPSC will be differentiated into hepatocytes (iHep). Since hepatocytes are the main producers of AAT, the iHep will be used to test different approaches of gene editing to correct various mutations. Gene editing will be conducted at University of Bern (Switzerland)

Gender: All

Ages: 18 Years - Any

Updated: 2025-03-24

1 state

Alpha1-antitrypsin Deficiency