U.S., March 25 -- ClinicalTrials.gov registry received information related to the study (NCT06892236) titled 'Preparation of IPSC for Cell Gene Editing for the Treatment of AATD' on Feb. 24.
Brief Summary: 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 (i...