Preface…
Table of Contents…
Contributing Authors…
1. Pathophysiology of Alpha-1 Antitrypsin Deficiency Liver Disease
JeffreyH. Teckman and Keith S. Blomenkamp
2. Pathophysiology of Alpha-1 Antitrypsin Lung Disease
Michael Kalfopoulos, Kaitlyn Wetmore, and Mai ElMallah
3. Measuring and Interpreting Serum AAT Concentration
Leslie Donato, Melissa Snyder, and Dina N. Greene
4. AAT Phenotype Identification by Isoelectric Focusing
Dina N. Greene, M.C. Elliot-Jelf, and David G. Grenache
5. Laboratory Diagnosis by Genotyping
Irene Belmonte, Luciana Montoto, and Francisco Rodríguez-Frías
6. Genotyping Protocol for the Alpha-1 Antitrypsin (PiZ) Mouse Model
Alisha M. Gruntman
7. Elastase-Induced Lung Emphysema Models in Mice
Béla Suki, Elzsébet Bartolák-Suki, and Patricia R.M. Rocco
8. Assessing Structure-Function Relations in Mice Using the Forced Oscillation Technique and Quantitative Histology
Harikrishnan Parameswaran and Béla Suki
9. 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
Airiel M. Davis, Kristen E. Thane, and Andrew M. Hoffman
10. Design, Cloning, and In Vitro Screening of Artificial miRNAs to Silence Alpha-1 Antitrypsin
Florie Borel and Christian Mueller
11. Methods to Identify and Characterize siRNAs Targeting Serpin A1 In Vitro and In Vivo using RNA Interference
Stuart Milstein, Kun Qian, Linying Zhang, and Alfica Sehgal
12. Knockdown of Z Mutant Alpha-1 Antitrypsin in Vivo Using Modified DNA Antisense Oligonucleotides
Mariam Aghajan, Shuling Guo, and Brett P. Monia
13. Immunohistochemistry Staining for Human Alpha-1 Antitrypsin
Dongtao A. Fu and Martha Campbell-Thompson
14. Periodic Acid-Schiff Staining with Diastase
Dongtao A. Fu and Martha Campbell-Thompson
15. Protocol for Directed Differentiation of Human Induced Pluripotent Stem Cells (iPSCs) to a Hepatic Lineage
Joseph E. Kaserman and Andrew A. Wilson
16. Isolation of Kupffer Cells and Hepatocytes from a Single Mouse Liver
Marcela Aparicio-Vergara, Michaela Tencerova, Cecilia Morgantini, Emelie Barreby, and Myriam Aouadi
17. Alpha-1 Antitrypsin Transcytosis and Secretion
Angelia D. Lockett
18. Measuring the Effect of Histone Deacetylase Inhibitors (HDACi) on the Secretion and Activity of Alpha-1 Antitrypsin
Chao Wang, Marion Bouchecareilh, and William E. Balch
19. Expression and Purification of Active Recombinant Human 1-Antitrypsin (AAT) from Escherichia coli
Beena Krishnan, Lizbeth Hedstrom, Daniel N. Hebert, Lila M. Gierasch, and Anne Gershenson
20. Quantification of Total Human Alpha-1 Antitrypsin by Sandwich ELISA
Qiushi Tang, Alisha M. Gruntman, and Terence R. Flotte
21. Quantification of Murine AAT by Direct ELISA
Andrew Cox and Christian Mueller
22. Quantification of Z-AAT by a Z-Specific “Sandwich” ELISA
Florie Borel, Qiushi Tang, and Christian Mueller
23. Semi-Quantitation of Monomer and Polymer Alpha-1 Antitrypsin by Centrifugal Separation and Assay by Western Blot of Soluble and Insoluble Components
Keith S. Blomenkamp and Jeffrey H. Teckman
24. Electrophoresis- and FRET-Based Measures of Serpin Olymerization
Sarah V. Faull, Anwen E. Brown, Imran Haq, and James A. Irving
25. Therapeutics: Alpha-1 Antitrypsin Augmentation Therapy
Michael Campos and Jorge Lascano
26. Therapeutic Option in Alpha-1 Antitrypsin Liver Transplantation
27. Therapeutics: Gene Therapy for Alpha-1 Antitrypsin Deficiency
Alisha M. Gruntman and Terence R. Flotte
