Alpha-1 Antitrypsin Deficiency

Pathophysiology of Alpha-1 Antitrypsin Deficiency Liver Disease.- Pathophysiology of Alpha-1 Antitrypsin Lung Disease.- Measuring and Interpreting Serum AAT Concentration.- AAT Phenotype Identification by Isoelectric Focusing.- Laboratory Diagnosis by Genotyping.- Genotyping Protocol for the Alpha-1 Antitrypsin (PiZ) Mouse Model.- Elastase-Induced Lung Emphysema Models in Mice.- Assessing Structure-Function Relations in Mice Using the Forced Oscillation Technique and Quantitative Histology.- Practical Methods for Assessing Emphysema Severity Based on Estimation of Linear Mean Intercept (Lm) in the Context of Animal Models of Alpha-1 Antitrypsin Deficiency.- Design, Cloning, and In Vitro Screening of Artificial miRNAs to Silence Alpha-1-Antitrypsin.- Methods to Identify and Characterize siRNAs Targeting Serpin A1 In Vitro and In Vivo using RNA Interference.- Knockdown of Z Mutant Alpha-1 Antitrypsin in Vivo Using Modified DNA Antisense Oligonucleotides.- Immunohistochemistry Staining for Human Alpha-1 Antitrypsin.- Periodic Acid-Schiff Staining with Diastase.- Protocol for Directed Differentiation of Human Induced Pluripotent Stem Cells (iPSCs) to a Hepatic Lineage.- Isolation of Kupffer Cells and Hepatocytes from a Single Mouse Liver.- Alpha-1 Antitrypsin Transcytosis and Secretion.- Measuring the Effect of Histone Deacetylase Inhibitors (HDACi) on the Secretion and Activity of Alpha-1 Antitrypsin.- Expression and Purification of Active Recombinant Human 1-Antitrypsin (AAT) from Escherichia coli.- Quantification of Total Human Alpha-1 Antitrypsin by Sandwich ELISA.- Quantification of Murine AAT by Direct ELISA.- Quantification of Z-AAT by a Z-Specific “Sandwich” ELISA.- Semi-Quantitation of Monomer and Polymer Alpha-1 Antitrypsin by Centrifugal Separation and Assay by Western Blot of Soluble and Insoluble Components.- Electrophoresis- and FRET-Based Measures of Serpin Olymerization.- Therapeutics: Alpha-1 Antitrypsin Augmentation Therapy.- Therapeutic Option in Alpha-1 Antitrypsin Liver Transplantation.- Therapeutics: Gene Therapy for Alpha-1 Antitrypsin Deficiency.