- What is the natural disease course, and what factors determine disease progression?- What are the biological mechanisms underlying pediatric and adult AATD-related liver disease?- What endpoints should be used in clinical trials?- What are the “toxic species” in AATD? (ie, the molecular consequences of different AAT assemblies)- What is the best model to study AATD-associated liver disease mechanisms?- Do we need AATD-specific questionnaires to evaluate disease burden? The gene discussed is SERPINA1; the disease is liver disorder.