This study focuses on generating induced pluripotent stem cells (iPSC) for cell gene editing aimed at treating alpha-1 antitrypsin deficiency (AATD). Adults aged 18 and older with severe AATD carrying two disease-causing variants may take part. Conducted by Fondazione IRCCS Policlinico San Matteo di Pavia, this study without an assigned phase is enrolling participants by invitation.
The summary above is a plain-language rendering of the official record. The original English title is shown for reference.
Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.
Inclusion Criteria: * age ≥ 18 years at the time of signing the ICF * diagnosis of severe AATD (2 pathological variants) Exclusion Criteria: * incapability to give informed consent * subject under augmentation therapy