Appendix 1
Year Investigator Basic Science Clin. Science Treatment Epi./Social Detection Lung Liver
1999 Song x   x     x  
1999 Fleming         x    
1999 Mullins       x   x x
1999 Guaressimov x         x  
2000 Hidvegi x           x
2000 Seifers x           x
2000 Anthony x   x     x x
2000 Teckman              
2000 Zern x   x     x x
2001 Seifers x           x
2001 Coors       x      
2001 Merz       x      
2001 Miravitiles         x    
2001 Cox   x          
2001 De Serres       x      
2001 Stoller   x x x   x  
2001 Buchanan   x   x      
2001 Cannon   x   x      
2001 Brenner x   x       x
2001 Pearlmutter x           x
2001 Brantly   x x     x x
2001 Flotte   x x     x  
2001 Luke x         x  
2002 Arvan x           x
2002 Zern x           x
2002 Teckman x           x
2002 Wachbroidt   x   x   x  
2002 Spencer x         x  
2002 Schiff   x         x
2002 Cosio x         x  
2002 McElvaney x         x  
2002 Mergo   x       x  

2003

Clayton       x      
2003 Fanos       x   x  
2003 Lee x            
2003 Sifers x           x
2003 Tuder x         x  
2003 Welsh x   x     x  
2004 Aulak x         x x
2004 Berclaz x   x     x  
2004 Bezerra   x         x
2004 Brantly x           x
2004 Conkright x         x x
2004 D'Armiento x         x  
2004 Greene x         x  
2004 Guerra   x       x  
2004 Liu x           x
2004 Mahadeva x         x  
2004 Miki x   x       x
2004 Miravitiles         x    
2004 Petrache x         x  
2004 Shaker   x       x  
2004 Sifers x           x
2004 Vedal   x   x      
2004 Verfaille x   x       x
2004 Wagner   x x        
2004 Zhou x         x x
2005 Brantly x           x
2005 Campos         x    
2005 Cantor x   x     x  
2005 DeMeo   x       x  
2005 Ford x     x      
2005 Houghton x         x x
2005 Marszal x         x x
2005 Sifers x           x
2005 Taggart x         x  
2005 Teckman x   x       x
2005 Zern x   x       x
2006 Hersh   x       x  
2006 Kharadman x x       x  
2006 Shao x         x  
2006 Shifren x         x  
2006 Steer x   x       x
2006 Yu x   x     x  
2007 Sifers x           x
2007 McElvaney   x   x   x  
2007 Olson   x x     x  
2007 Grabowski x   x       x
2007 Glenn x           x
2007 Gildea   x x x   x  
2008 Campos   x x        
2008 Chatburn       x      
2008 Coors       x      
2008 DeMeo   x   x      
2008 Flotte x           x
2008 Greene x           x
2008 Holm       x      
2008 Jun x         x  
2008 Krishnan x         x x
2008 Li x   x       x
2008 Liu x   x       x
2008 Lougee         x    
2008 Sharma x   x       x
2008 Steffen x         x x
2008 Tuder x         x  
Totals   55 22 24 18 3 44 40

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Appendix 2

Alpha-1 Related Publications by A1F Funded Investigators

Abusriwil, Hatem
Alpha-1 Antitrypsin replacement therapy: current status
Current Opinions in Pulmonary Medicine

Adair-Kirk, Tracy
Neutrophil elastase cleaves Laminin-332 (laminin-5) generating peptides that are chemotactic for neutrophils
Journal of Biological Chemistry

Arvan, Peter
Endoplasmic reticulum (ER) chaperone regulation and survival of cells compensating for deficiency in the ER stress response kinase, PERK
Journal of Biological Chemistry

The trafficking of alpha-1 antitrypsin, a post-Golgi secretory pathway marker, in INS-1 pancreatic beta cells
Journal of Biological Chemistry

Bezerra, Jorge
High Prevalence of alpha-1 antitrypsin heterozygosity in children with chronic liver disease
Journal of Pediatric Gastroenterology

Hepatocyte transplantation.
Advancing Biology and treating children Clinics in Liver Disease

Blumental-Perry, Anna
Phosphatidylinositol 4-phosphate formation at ER exit sites regulates ER export
Development Cell

Bowler, Russell
Cigarette smoke impairs clearance of apoptotic cells through oxidant-dependent activation of RhoA
American Journal of Repiratory Care Medicine

Lovastatin enhances clearance of apoptotic cells (efferocytosis) with implications for chronic obstructive pulmonary disease
The Journal of Immunology

Brantly, Mark
Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1-AAT gene therapy
Proceedings of the National Academy of Sciences

Single nucleotide polymorphism-mediated translational suppression of endoplasmic reticulum mannosidase 1 modifies the onset of end-stage liver disease in Alpha-1 Antitrypsin Deficiency
Hepatology

Heritability of lung function in severe Alpha-1 Antitrypsin Deficiency
Human Heredity

Human alpha-1 antitrypsin therapy induces fatal anaphylaxis in non-obese diabetic mice
Clinical and Experimental Immunology

Endothelial alpha-1 antitrypsin attenuates cigarette smoke induced apoptosis in vitro
COPD

Alpha-1 Antitrypsin aerosolized augmentation abrogates neutrophil elastase-induced expression of cathepsin B and matrix metalloprotease 2 in vivo and in vitro
Thorax

Primary care diagnosis of Alpha-1 Antitrypsin Deficiency: issues and opportunities
Cleveland Clinic Journal of Medicine

Prevalence of Alpha-1 Antitrypsin Deficiency in poorly controlled asthma-results from the ALA-ACRC low-dose theophylline trial
Journal of Asthma

IL10 polymorphisms are associated with airflow obstruction in severe Alpha-1 Antitrypsin Deficiency
American Journal of Respiratory Cell and Molecular Biology

In vivo post-transcriptional gene silencing of alpha-1 antitrypsin by adeno-associated virus vectors expressing siRNA
Laboratory Investigation

Determinants of airflow obstruction in severe Alpha-1 Antitrypsin Deficiency
Thorax

Multi-Center study: the biochemical efficacy, safety and tolerability of a new alpha-1 proteinase inhibitor, Zemaira
COPD

Efficient and accurate approaches to the laboratory diagnosis of Alpha-1 Antitrypsin Deficiency: The promise of early diagnosis and intervention
Clinical Chemistry

Phase 1 trial of intramuscular injection of a recombinant adeno-associated virus serotype 2 alpha-1 antitrypsin (AAT) vector in AAT deficient adults
Human Gene Therapy

Does the heterozygous state of Alpha-1 Antitrypsin Deficiency have a role in chronic liver diseases? Interim results of a large case-control study
Journal of Pediatric Gastroenterology

Genetic testing of minors for Alpha-1 Antitrypsin Deficiency
Archives of Pediatrics and Adolescent Medicine

Cytotoxic concentrations of alpha-defensins in the lungs of individuals with Alpha-1 Antitrypsin Deficiency and moderate to severe lung disease
Cytokine

Antibody response to aerosolized transgenic human alpha-1 antitrypsin
New England Journal of Medicine

Genetic testing for Alpha-1 Antitrypsin Deficiency
Genetics in Medicine

Z alpha-1 antitrypsin polymerizes in the lung and acts as a neutrophil chemoattractant
CHEST

Phase 1 trial of intramuscular injection of a recombinant adeno-associated virus alpha-1 antitrypsin (rAAV2-CB-hAAT) gene vector to AAT deficient adults
PMID

Role of human neutrophil peptides in lung inflammation associated with Alpha-1 Antitrypsin Deficiency
Lung Cellular and Molecular Physiology

Asthma features in severe Alpha-1 Antitrypsin Deficiency: experience of the National Heart, Lung, and Blood Institute Registry
CHEST

Alpha-1 antitrypsin: not just an antiprotease: extending the half-life of a natural anti-inflammatory molecule by conjugation with polyethylene glycol
American Journal of Respiratory Cell and Molecular Biology

Biochemical efficacy and safety of a new pooled human plasma alpha-1 antitrypsin, Respitin
CHEST

Formation and current results of a patient-organized registry for Alpha-1 Antitrypsin Deficiency
CHEST

Molecular mechanisms of alpha-1 antitrypsin null alleles
Respiratory Medicine

Lung neutrophil burden correlates with increased pro-inflammatory cytokines and decreased lung function in individuals with Alpha-1 Antitrypsin Deficiency
PMID

Endothelial nitric oxide synthase as a potential susceptibility gene in the pathogenesis of emphysema in Alpha-1 Antitrypsin Deficiency
American Journal of Respiratory Cell and Molecular Biology

Efficient in vivo catheter-based pericardial gene transfer mediated by adenoviral vectors
Clinical Cardiology

Brenner, David
Genomics of liver fibrosis and cirrhosis
Seminars in Liver Disease

Genetic polymorphisms and the progression of liver fibrosis: a critical appraisal
Hepatology

Brodsky, Jeff
One step at a time: endoplasmic reticulum-associated degradation
Nature Reviews Molecular Cellular Biology

The protective and destructive roles played by molecular chaperones during ERAD
Biochemical Journal

Molecular pathogenesis of Alpha-1 Antitrypsin Deficiency associated liver disease: a meeting review
Hepatology

Characterization of an ERAD gene as VPS30/ATG6 reveals two alternative and functionally distinct protein quality control pathways: one for soluble Z variant of human alpha-1 porteinase inhibitor (A1 PiZ) and another for aggregates of A1 PiZ
Molecular Biology of the Cell

Yeast as a model system to investigate protein conformational diseases
Methods in Molecular Biology

ADD66, a gene involved in the endoplasmic reticulum-associated degradation of alpha-1 antitrypsin Z in yeast, facilitates proteasome activity and assembly
Molecular Biology of the Cell

Buchanan, Robert
Analysis of male residents in community nursing facilities: comparisons of Veterans Health Administration residents to other residents
Journal of Rehabilitation Research Development

Campos, Michael
Safety and efficacy of alpha-1 antitrypsin augmentation therapy in the treatment of patients with Alpha-1 Antitrypsin Deficiency
Biologics

Exacerbations in subjects with Alpha-1 Antitrypsin Deficiency receiving augmentation therapy
Respiratory Medicine

Effects of a disease management program in individuals with Alpha-1 Antitrypsin Deficiency
COPD

Clinical characteristics of subjects with symptoms of Alpha-1 Antitrypsin Deficiency older than 60 years
CHEST

Influenza vaccination in subjects with Alpha-1 Antitrypsin Deficiency
CHEST

Trends in the diagnosis of symptomatic patients with Alpha-1 Antitrypsin Deficiency between 1968 and 2003
CHEST

Cantor, Jerome
Synergistic effect of hydrogen peroxide and elastase on elastic fiber injury in vitro
Experimental Biology and Medicine

Aerosolized hyaluronan limits airspace enlargement in a mouse model of cigarette smoke-induced pulmonary emphysema
Experimental Lung Research

Carroll, Tomas Selenoprotein S/SEPS1 modifies endoplasmic reticulum stress in Z variant Alpha-1 Antitrypsin Deficiency
Journal of Biological Chemistry

Viral inhibition of IL-1 and neutrophil elastase-induced inflammatory responses in bronchial epithelial cells
Journal of Immunology

Neutrophil elastase up-regulates interleukin-8 via toll-like receptor 4
FEBS Letters

Coors, Marylin
Anxiety related to genetic testing for Alpha-1 Antitrypsin Deficiency and cystic fibrosis in COPD and/or bronchiectasis
Community Genetics

Cosio, Manuel
Alpha-1 antitrypsin determines the pattern of emphysema and function in tobacco smoke-exposed mice: parallels with human disease
American Journal of Respiratory and Critical Care Medicine

Cox, Diane
Prenatal diagnosis for Alpha-1 Antitrypsin Deficiency
Prenatal Diagnosis

Coxson, Harvey
Quantitative chest tomography in COPD research: a chairman's summary
Proceedings of the American Thoracic Society

Cruz, Pedro
Recombinant AAV serotype and capsid mutant comparison for pulmonary gene transfer of alpha-1 antitrypsin using invasive and noninvasive deliver
Molecular Therapy

Removal of envelope protein-free retroviral vectors by anion-exchange chromatography to improve product quality
Journal of Separation Science

The promise of gene therapy for the treatment of Alpha-1 Antitrypsin Deficiency
Pharmacogenomics

In vivo post-transcriptional gene silencing of alpha-1 antitrypsin by adeno-associated virus vectors expressing siRNA
Laboratory Investigation

Scaleable purification process for gene therapy retroviral vectors
Journal of Genetic Medicine

Alpha-1 antitrypsin inhibits caspase-3 activity, preventing lung endothelial cell apoptosis
American Journal of Pathology

Purification of retroviral vectors for clinical application: biological implications and technological challenges
Journal of Biotechnology

A novel antiapoptotic role for alpha-1 antitrypsin in the prevention of pulmonary emphysema
American Journal of Respiratory and Critical Care Medicine

D'Armiento, Jeanine
Transgenic expression of matrix metalloproteinase-9 causes adult-onset emphysema in mice associated with the loss of alveolar elastin
American Journal of Lung Cellular and Molecular Physiology

De Serres, Fred
Long-term augmentation therapy with alpha-1 antitrypsin in an MZ-AAT severe persistent asthma
Monaldi Archives of Chest Disease

PI S and PI Z Alpha-1 Antitrypsin Deficiency worldwide. A review of existing genetic epidemiological data
Monaldi Archives of Chest Disease

Estimating the risk for Alpha-1 Antitrypsin Deficiency among COPD patients: evidence supporting targeted screening
COPD

Estimated numbers and prevalence of PI S and PI Z alleles of Alpha-1 Antitrypsin Deficiency in Asia
European Respiratory Journal

Estimated numbers and prevalence of PI S and PI Z alleles of Alpha-1 Antitrypsin Deficiency in European countries
European Respiratory Journal

Alpha-1 Antitrypsin Deficiency in Italy: Regional differences of the PI S and PI Z deficiency alleles
Monaldi Archives of Chest Disease

High prevalence of alpha-1 antitrypsin phenotypes in viral hepatitis B infected patients in Iran
Hepatology Research

Alpha-1 antitrypsin phenotypes and HLA-B27 typing in uveitis patients in southeast Iran
Clinical Biochemistry

Health implications of Alpha-1 Antitrypsin Deficiency in Sub-Sahara African countries and their emigrants in Europe and the New World
Genetics Medicine

Alpha-1 antitrypsin and fibromyalgia: new data in favor of the inflammatory hypothesis of fibromyalgia
Medical Hypothesis

PI S and PI Z Alpha-1 Antitrypsin Deficiency: estimated prevalence and number of deficient alleles in Spain
Medicina Clinica

Alpha-1 antitrypsin replacement therapy controls fibromyalgia symptoms in 2 patients with PI ZZ Alpha-1 Antitrypsin Deficiency
Journal of Rheumatology

Alpha-1 Antitrypsin Deficiency is not a rare disease but a disease that is rarely diagnosed
Environmental Health

Perspectives Genetic epidemiology of Alpha-1 Antitrypsin Deficiency in North America and Australia/New Zealand: Australia, Canada, New Zealand and the United States of America
Clinical Genetics

Environmental, occupational, and genetic risk factors for Alpha-1 Antitrypsin Deficiency
Environmental Health

Perspectives Genetic epidemiology of Alpha-1 Antitrypsin Deficiency in southern Europe: France, Italy, Portugal and Spain
Clinical Genetics

Worldwide racial and ethnic distribution of Alpha-1 Antitrypsin Deficiency: summary of an analysis of published genetic epidemiologic surveys
CHEST

Deep-Sharma, Amar
Murine embryonic stem cell-derived hepatic progenitor cells engraft in recipient livers with limited capacity of liver tissue formation
Cell Transplantation

DeMeo, Dawn
Heritability of lung function in severe Alpha-1 Antitrypsin Deficiency
Human Heredity

Determinants of airflow obstruction in severe Alpha-1 Antitrypsin Deficiency
Thorax

The SERPINE2 gene is associated with chronic obstructive pulmonary disease
Proceedings of the American Thoracic Society

Predictors of survival in severe, early onset COPD
Chest

Alpha-1 Antitrypsin Deficiency 2: genetic aspects of Alpha-1 Antitrypsin Deficiency: phenotypes and genetic modifiers of emphysema risk
Thorax

Eissa, N. Tony
Endothelial nitric oxide synthase as a potential susceptibility gene in the pathogenesis of emphysema in Alpha-1 Antitrypsin Deficiency
American Journal of Respiratory Cell and Molecular Biology

Fanos, Joanna
The lion, the witch and the wardrobe: impact on siblings of individuals with AAT Deficiency
American Journal of Medical Genetics

Flotte, Terence
Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1-AAT gene therapy
Proceedings of the National Academy of Science

Recombinant AAV serotype and capside mutant comparison for pulmonary gene transfer of alpha-1 antitrypsin using invasive and noninvasive delivery
Molecular Therapy

The promise of gene therapy for the treatment of Alpha-1 Antitrypsin Deficiency
Pharmacogenomics

In vivo post-transcriptional gene silencing of alpha-1 antitrypsin by adeno-associated virus vectors expressing siRNA
Laboratory Investigation

Alpha-1 antitrypsin inhibits caspase-3 activity , preventing lung endothelial cell apoptosis
American Journal of Pathology

Therapeutic level of functional human alpha-1 antitrypsin (hAAT) secreted from murine muscle transduced by adeno-associated virus (rAAV1) vector
Journal of Genetics Medicine

A novel antiapoptotic role for alpha-1 antitrypsin in the prevention of pulmonary emphysema
American Journal of Respiratory and Critical Care Medicine

Adeno-associated virus-based gene therapy for inherited disorders
Pediatric Research

Recent developments in recombinant AAV-mediated gene therapy for lung diseases
Current Gene Therapy

Phase 1 trial of intramuscular injection of a recombinant adeno-associated virus alpha-1 antitrypsin (rAAV2-CB-hAAT) gene vector to AAT-deficient adults
Human Gene Therapy

Intramuscular administration of recombinant adeno-associated virus 2 alpha-1 antitrypsin (rAAV-SERPINA1) vectors in a nonhuman primate model: safety and immunologic aspects
Molecular Therapy

Recombinant adeno-associated virus gene therapy for cystic fibrosis and Alpha-1 Antitrypsin Deficiency
Chest

Gershenson, Anne
Short-lived protease serpin complexes: partial disruption of the rat trypsin active site
Protein Science

Conformational distributions of protease-serpin complexes: a partially translocated complex
Biochemistry

Gildea, Thomas
Lung volume reduction surgery in patients with emphysema and Alpha-1 Antitrypsin Deficiency
Annals of Thoracic Surgery

Cost effectiveness of population screening for Alpha-1 Antitrypsin Deficiency: a decisions analysis
COPD

Cost effectiveness analysis of augmentation therapy for severe Alpha-1 Antitrypsin Deficiency
American Journal of Respiratory and Critical Care Medicine

Emphysema in nonsmokers: Alpha-1 Antitrypsin Deficiency and other causes
Cleveland Clinic Journal of Medicine

Greene, Catherine
Gene targeted therapeutics for liver disease in Alpha-1 Antitrypsin Deficiency
Biologics

Selenoprotein S/SEPS1 modifies endoplasmic reticulum stress in Z variant Alpha-1 Antitrypsin Deficiency
Journal of Biological Chemistry

Z alpha-1 antitrypsin polymerizes in the lung and acts as a neutrophil chemoattractant
CHEST

Local impairment of anti-neutrophil elastase capacity in community-acquired pneumonia
Journal of Infectious Diseases

Guerra, Stefano
A longitudinal study of alpha-1 antitrypsin phenotypes and decline in FEV1 in a community population
CHEST

Hersh, Craig
A genome-wide association study in chronic obstructive pulmonary disease: identification of two major susceptibility loci
PLOS Genetics

Analysis of exonic elastin variants in severe, early-onset chronic obstructive pulmonary disease
American Journal of Respiratory Cell and Molecular Biology

Genetic Linkage and association analysis of COPD-related traits on chromosome 8p
COPD

Predictors of survival in severe, early-onset COPD
CHEST

Hidvegi, Tunda
Regulator of G Signaling 16 is a marker for the distinct endoplasmic reticulum stress state associated with aggregated mutant alpha-1 antitrypsin Z in the classical form of Alpha-1 Antitrypsin Deficiency
Journal of Biological Chemistry

Intracellular inclusions containing mutant alpha-1 antitrypsin Z are propagated in the absence of autophagic activity
Journal of Biologic Chemistry

Accumulation of mutant alpha-1 antitrypsin Z in the endoplasmic reticulum activates caspases-4 and 12, NFkappaB, and BAP31 but not the unfolded protein response
Journal of Biologic Chemistry

Hoshino, Yuma
The development of emphysema in cigarette smoke-exposed mice is strain dependent
American Journal of Respiratory and Critical Care Medicine

Houghton, A. McGarry
Epithelial cell apoptosis causes acute lung injury masquerading as emphysema
American Journal of Respiratory Cell and Molecular Biology

Janciauskiene, Sabina
Oxidized alpha-1 antitrypsin stimulates the release of monocyute chemotactic protein-1 from lung epithelial cells: potential role in emphysema
American Journal of Lung Cellular and Molecular Physiology

Long-term augmentation therapy with alpha-1 antitrypsin in MZ-AAT severe persistent emphysema
Monaldi Archives of Chest Disease

New Findings in PiZZ Alpha-1 Antitrypsin Deficiency related panniculitis. Demonstration of skin polymers and high dosing requirements of intravenous augmentation therapy
Dermatology

Alpha-1 antitrypsin inhibits epithelial Na+ transport in vitro and in viv
American Journal of Respiratory Cell and Molecular Biology

An association between Type 2 diabetes and Alpha-1 Antitrypsin Deficiency
Diabetes Medicine

Alpha-1 antitrypsin inhibits the activity of the matriptase catalytic domain in vitro
American Journal of Respiratory Cell and Molecular Biology

Screening for Alpha-1 Antitrypsin Deficiency in Lithuanian patients with COPD
Respiratory Medicine

Endotoxin receptor CD14 in PiZ in Alpha-1 Antitrypsin Deficiency individual
Respiratory Research

Hereditary Alpha-1 Antitrypsin Deficiency can cause COPD. Substitution treatment seems to result in fewer infections and prolonged survival
Lakartidningen

TNF-alpha induced self expression in human lung endothelial cells is inhibited by native and oxidized alpha-1 antitrypsin
International Journal

Alpha-1 antitrypsin regulates CD14 expression and soluble CD14 levels in human monocytes in vitro
International Journal of Biochemistry and Cell Biology

Alpha-1 antitrypsin, old dog, new tricks. Alpha-1 antitrypsin exerts in vitro anti-inflammatory activity in human monocytes by elevating cAMP
Journal of Biological Chemistry

Plasma levels of alpha-1 antichymotrypsin and secretory leukocyte proteinase inhibitor in healthy and chronic obstructive pulmonary disease subjects with and without severe Alpha-1 Antitrypsin Deficiency
BMC Pulmonary Medicine

Do native and polymeric alpha-1 antitrypsin activate human neutrophils in vitro?
CHEST

C-36 peptide, a degradation product of alpha-1 antitrypsin, modulates human monocyte activation through LPS signaling pathways
International Journal of Biochemistry and Cell Biology

Alpha-1 antitrypsin inhibits Moraxella catarrhalis MID protein-induced tonsillar B cell proliferation and IL-6 release
Immunology Letters

Polymers of Z alpha-1 antitrypsin co-localize with neutrophils in emphysematous alveoli and are chemotactic in vivo
American Journal of Pathology

Polymerized alpha antitrypsin is present on lung vascular endothelium. New insights into the biological significance of alpha antitrypsin polymerization
Histopathology

ELISA for specific detection of PiZ-related Alpha-1 Antitrypsin Deficiency
Clinical Chemistry

Alpha-1 antitrypsin and its C-terminal fragment attenuate effects of degranulated neutrophil-conditioned medium on lung cancer HCC cells, in vitro
Cancer Cell International

Differential detection of PAS-positive inclusions formed by the Z, Siiyama, and Mmalton variants of alpha-1 antitrypsin
Hepatology

Inhibition of lipopolysaccharide-mediated human monocyte activation, in vitro by alpha-1 antitrypsin
Biochemical and Biophysical Research Communications

Concentration-dependent effects of native and polymerized alpha-1 antitrypsin on primary human monocytes, in vitro
BMC Cell Biology

Increased plasma levels of serine proteinase inhibitors in lung cancer patients
Anticancer Research

Divergent effects of alpha-1 antitrypsin on neutrophil activation in vitro
Biochemical and Biophysical Research Communications

Multiple effects of alpha-1 antitrypsin on breast carcinoma MDA-MB 468 cell growth and invasiveness
European Journal of Cancer Prevention

Detection of circulating endothelial cell polymers of Z and wild type alpha-1 antitrypsin by a monoclonal antibody
Journal of Biological Chemistry

Fibrillogenic C-terminal fragment of alpha-1 antitrypsin activates human monocytes via oxidative mechanisms
Cell and Tissue Research

Conformational properties of serine proteinase inhibitors (serpins) confer multiple pathophysiological roles
International Journal of Biochemistry, Biophysics and molecular biology

Alpha-1 antitrypsin is present in the specific granules of human eosinophilic granulocytes
Clinical and Experimental Allergy

Atherogenic properties of human monocytes induced by the carboxyl terminal proteolytic fragment of alpha-1 antitrypsin
Atherosclerosis

Human monocyte activation by cleaved form of alpha-1 antitrypsin involvement in the phagocytic pathway
European Journal of Biochemistry

Effects of fibrillar C-terminal fragment of cleaved alpha-1 antitrypsin on cholesterol homeostasis in HepG2 cells
Hepatology

Kheradmand, Farrah A protease-activated pathway underlying Th cell type 2 activation andallergic lung disease
Journal of Immunology

Kotton, Darrell
Sustained expression of alpha-1 antitrypsin after transplantation of manipulated hematopoietic stem cells
American Journal of Respiratory Cell Molecular Biology

Li, Chengwen
Therapeutic level of functional human alpha-1 antitrypsin (hAAT) secreted from murine muscle transduced by adeno-associated virus (rAAV1) vector
Journal of Gene Medicine

Luke, Cliff
Identification and activity of a lower eukaryotic serine proteinase inhibitor (serpin) from Cyanea capillata:
Biochemistry

Mahadeva, Ravi
Oxidized alpha-1 antitrypsin stimulates the release of monocyte chemotactic protein-1 from lung epithelial cells: potential role in emphysema
American Journal of Lung Cellular and Molecular Physiology

Small-molecule peptides inhibit Z alpha-1 antitrypsin polymerization
Journal of Cellular and Molecular Medicine

An association between Type 2 diabetes and Alpha-1 Antitrypsin Deficiency
Diabetic Medicine

Identification of a 4-mer peptide inhibitor that effectively blocks the polymerization of pathogenic Z alpha-1 antitrypsin
American Journal of Respiratory Cell and Molecular Biology

Polymers of Z alpha-1 antitrypsin co-localize with neutrophils in emphysematous alveoli and are chemotactic in vivo
American Journal of Pathology

Inhibiting polymerization: new therapeutic strategies for Z alpha-1 antitrypsin related emphysema
American Journal of Cell and Molecular Biology

Targeting a surface cavity of alpha-1 antitrypsin to prevent conformational disease
Journal of Biological Chemistry

Alpha-1 Antitrypsin Deficiency, liver disease and emphysema International
Journal of Biochemistry and Cell Biology

Alpha-1 antitrypsin polymerization and the serpinopathies: pathobiology and prospects for therapy
Journal of Clinical Investigation

Polymers of alpha-1 antitrypsin are chemotactic for human neutrophils: a new paradigm for the pathogenesis of emphysema
American Journal of Respiratory Cell and Molecular Biology

6-mer peptide selectively anneals to a pathogenic serpin conformation and blocks polymerization. Implications for the prevention of Z alpha-1 antitrypsin related cirrhosis
Journal of Biological Chemistry

Characterization of a new variants of alpha-1 antitrypsin E (Johannesburg) (H15N) in association with asthma
Human Mutation

A novel polymorphism (471CT) in alpha-1 antitrypsin in a patient with asthma
Human Mutation

Association of alpha-1 antichymotrypsin deficiency with milder lung disease in patients with cystic fibrosis
Thorax

McElvaney, Noel Gerald
Gene targeted therapeutics for liver disease in Alpha-1 Antitrypsin Deficiency
Biologics

Selenoprotein S/SEPS1 modifies endoplasmic reticulum stress in Z variant Alpha-1 Antitrypsin Deficiency
Journal of Biological Chemistry

Heritability of lung function in severe Alpha-1 Antitrypsin Deficiency
Human Heredity

Alpha-1 antitrypsin aerosolized augmentation abrogates neutrophil elastase-induced expression of Cathepsin B and matrix metalloprotease 2 in vivo and in vitro
Thorax

Alpha-1 Antitrypsin Deficiency: a conformational disease associated with lung and liver manifestations
Journal of Inherited Metabolic Disease

IL10 polymorphisms are associated with airflow obstruction in severe Alpha-1 Antitrypsin Deficiency
American Journal of Respiratory Cell and Molecular Biology

Alpha-1 Antitrypsin Deficiency: current concepts
Lung

Determinants of airflow obstruction in severe Alpha-1 Antitrypsin Deficiency
Thorax

Alpha-1 Antitrypsin Deficiency and computed tomography findings
Journal of Computer Assisted Tomography

Z alpha-1 antitrypsin polymerizes in the lung and acts as a neutrophil chemoattractant
CHEST

Anti-proteinase 3 antibody activation of neutrophils can be inhibited by alpha-1 antitrypsin
American Journal of Respiratory Cell and Molecular Biology

Alpha-1 Antitrypsin Deficiency: biological answers to clinical questions
The American Journal of the Medical Sciences

Alpha-1 Antitrypsin Deficiency
Irish Medical Journal

Merz, Jon
Ethical issues in identifying and recruiting participants for familial genetic research
American Journal of Medical Genetics

Protecting subjects' interest in genetics research
American Journal of Human Genetics

Miki, Toshio
Quantitative comparison of stem cell marker-positive cells in fetal and term human amnion
Journal of Reproductive Immunology

Production of hepatocyte-like cells from human amnion
Methods in Molecular Biology

Isolation of amniotic epithelial stem cells
Current Protocols in Stem Cell Biology

Hepatocyte transplantation: clinical experience and potential for future use
Cell Transplantation

Miravitlles, Marc
Augmentation therapy for emphysema due to Alpha-1 Antitrypsin Deficiency
Therapeutic Advances in Respiratory Diseases

Alpha-1 Antitrypsin Deficiency: diagnosis and treatment
Journal de Brasileira de Pneumologia

Development and results of the Spanish registry of patients with Alpha-1 Antitrypsin Deficiency
International Journal of Chronic Obstructive Pulmonary Disease

Guidelines for the diagnosis and management of Alpha-1 Antitrypsin Deficiency
Archivos de Bronchoneumologica

Alpha-1 Antitrypsin Deficiency: situation in Spain and development of a screening program
Archivos de Bronchoneumologica

Emphysema due to Alpha-1 Antitrypsin Deficiency: familial study of the YBarcelona variant
CHEST

Rapid screening for Alpha-1 Antitrypsin Deficiency in patients with chronic obstructive pulmonary disease using dried blood specimens
American Journal of Respiratory and Critical Care Medicine

Mullins, Daniel
Major components of the direct medical costs of Alpha-1 Antitrypsin Deficiency
CHEST

Olson, Eric L
Genetic modifiers of liver disease in cystic fibrosis
Journal of the American Medical Association

Perlmutter, David
Regulator of G signaling 16 is a marker for the distinct endoplasmic reticulum stress state associated with aggregated mutant alpha-1 antitrypsin Z in the classical form of Alpha-1 Antitrypsin Deficiency
Journal of Biological Chemistry

ADD66, a gene involved in the endoplasmic reticulum-associated degradation of alpha-1 antitrypsin Z in yeast, facilitates proteasome activity and assembly
Molecular Biology of the Cell

Molecular pathogenesis of Alpha-1 Antitrypsin Deficiency associated liver disease: a meeting review
Hepatology

The role of autophagy in Alpha-1 Antitrypsin Deficiency: a specific cellular response in genetic diseases associated with aggregation-prone proteins
Autophagy

Pathogenesis of chronic liver injury and hapatocellular carcinoma in Alpha-1 Antitrypsin Deficiency
Pediatric Research

Intracellular inclusions containing mutant alpha-1 antitrypsin Z are propagated in the absence of autophagic activity
Journal of Biological Chemistry

Accumulation of mutant alpha-1 antitrypsin Z in the endoplasmic reticulum activates caspases-4 and 12, NFkappaB, and BAP31 but not the unfolded protein response
Journal of Biologic Chemistry

Alpha-1 Antitrypsin Deficiency: a new paradigm for hepatocellular carcinoma in genetic liver disease
Hepatology

Grp78, Grp94, and Grp170 interact with alpha-1 antitrypsin mutants that are retained in the endoplasmic reticulum
American Journal of Gastrointestinal and Liver Physiology

Alpha-1 Antitrypsin Deficiency: diagnosis and treatment
Clinical Liver Disease

Analyses of hepatocellular proliferation in a mouse model of Alpha-1 Antitrypsin Deficiency
Hepatology

Alpha-1 Antitrypsin Deficiency: liver disease associated with retention of a mutant secretory glycoprotein in the endoplasmic reticulum
Methods in Molecular Biology

Liver Injury in Alpha-1 Antitrypsin Deficiency: an aggregated protein induces mitochondrial injury
Journal of Clinical Investigation

Chemical chaperones: a pharmacological strategy for disorders of protein folding and trafficking
Pediatric Research

The cellular response to aggregated proteins associated with human disease
Journal of Clinical Investigation

Fasting in alpha-1 antitrypsin deficient liver: constitutive activation of autophagy
American Journal of Gastrointestinal and Liver Physiology

Cell-specific involvement of HNF-1 beta in alpha-1 antitrypsin gene expression in human respiratory epithelial cells
American Journal of Lung Cellular and Molecular Physiology

The proteasome participates in degradation of mutant alpha-1 antitrypsin Z in the endoplasmic reticulum of hepatoma-derived hepatocytes
Journal of Biological Chemistry

A naturally occurring nonpolymerogenic mutant of alpha-1 antitrypsin characterized by prolonged retention in the endoplasmic reticulum
Journal of Biological Chemistry

Liver injury in Alpha-1 Antitrypsin Deficiency
Clinical Liver Disease

Alpha-1 Antitrypsin Deficiency
Current Treatment Options in Gastroenterology

Retention of mutant alpha-1 antitrypsin Z in endoplasmic reticulum is associated with an autophagic response
American Journal of Gastrointestinal and Liver Physiology

Alpha-1 Antitrypsin Deficiency associated liver disease progresses slowly in some children
Journal of Pediatric Gastroenterology and Nutrition

Circulating serpin tumor markers SCCA1 and SCCA2 are not actively secreted but reside in the cytosol of squamous carcinoma cells
International Journal of Cancer

Chemical chaperones mediate increased secretion of mutant alpha-1 antitrypsin Z: A potential pharmacological strategy for prevention of liver injury and emphysema in Alpha-1 Antitrypsin Deficiency
Proceedings of the National Academy of Sciences

Role of ubiquitin in proteasomal degradation of mutant alpha-1 antitrypsin Z in the endoplasmic reticulum
American Journal of Gastrointestinal and Liver Physiology

Glucosidase and mannosidase inhibitors mediate increased secretion of mutant alpha-1 antitrypsin Z
Journal of Biological Chemistry

Misfolded proteins in the endoplasmic reticulum
Laboratory Investigation

Regulation of alpha-1 antitrypsin gene expression in human intestinal epithelial cell line caco-2 by HNF-1 alpha and HNF-4
American Journal of Physiology

Petrache, Irina
Safety and efficacy of alpha-1 antitrypsin augmentation therapy in the treatment of patients with Alpha-1 Antitrypsin Deficiency
Biologics

Mechanisms of alpha-1 antitrypsin endocytosis by lung endothelium
FASEB

CFTR regulation of intracellular pH and ceramides is required for lung endothelial cell apoptosis
American Journal of Respiratory Cell and Molecular Biology

Spinal ceramide modulates the development of morphine antiociceptive tolerance via peroxynitrite-mediated nitroxidative stress and neuroimmune activation
Journal of Pharmacology Experimental Therapy

Targeted induction of lung endothelial cell apoptosis causes emphysema-like changes in the mouse
Journal of Biological Chemistry

Alpha-1 antitrypsin inhibits caspase-3 activity, preventing lung endothelial cell apoptosis
American Journal of Pathology

A novel antiapoptotic role for alpha-1 antitrypsin in the prevention of pulmonary emphysema
American Journal of Respiratory Critical Care Medicine

Schiff, Eugene
Does the Heterozygous State of Alpha-1 Antitrypsin Deficiency Have a Role in Chronic Liver Diseases? Interim Results of a Large Case-Control Study
Journal of Pediatric Gastroenterology and Nutrition

Shaker, Saher
Quantitative assessment of emphysema distribution in smokers and patients with Alpha-1 Antitrypsin Deficiency
Respiratory Medicine

Shao, Matt
Neutrophil elastase induces MUC5AC mucin production in human airway epithelial cells via a cascade involving protein kinase C, reactive oxygen species and TNF-alpha-converting enzyme
Journal of Immunology

Sharp, Richard
Responsible patient advocacy: perspectives from the Alpha-1 Foundation
American Journal of Medical Genetics

Environmental, occupational and genetic risk factors for Alpha-1 Antitrypsin Deficiency
Environmental Health Perspectives

Shifren, Adrian
Elastin protein levels are a vital modifier affecting normal lung development and susceptibility to asthma
American Journal of Lung Cellular and Molecular Physiology

Sifers, Richard
Single nucleotide polymorphism-mediated translational suppression of endoplasmic reticulum mannosidase 1 modifies the onset of end-stage liver disease in Alpha-1 Antitrypsin Deficiency
Hepatology

The mammalian UPR boosts glycoprotein ERAD by suppressing the proteolytic downregulation of ER mannosidase 1
Journal of Cell Science

Small molecules block the polymerization of Z alpha-1 antitrypsin and increase clearance of intracellular aggregates
Journal of Medicinal Chemistry

Human endoplasmic reticulum mannosidase 1 is subject to regulated proteolysis
Journal of Biological Chemistry

Alpha-1 antitrypsin as a model to assess glycan function in endoplasmic reticulum
Methods

Human EDEM2, a novel homolog of family 47 glycosidases, is involved in ER-associated degradation of glycoproteins
Glycobiology

Elucidation of the molecular logic by which misfolded alpha-1 antitrypsin is preferentially selected for degradation
Proceedings of the National Academy of Sciences

Organizational diversity among distinct glycoprotein endoplasmic reticulum-associated degradation programs
Molecular Biology of the Cell

Spencer, Terry
Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1-AAT gene therapy
Proceedings of the National Academy of Sciences

Alpha-1 antitrypsin protects beta cells from apoptosis
Diabetes

Phase 1 trial of intramuscular injection of a recombinant adeno-associated virus serotype 2 alpha-1 antitrypsin (AAT) vvector in AAT-deficient adults
Human Gene Therapy

Antibody response to aerosolized transgenic human alpha-1 antitrypsin New England
Journal of Medicine

Role of human neutrophil peptides in lung inflammation associated with Alpha-1 Antitrypsin Deficiency
American Journal of Lung Cellular and Molecular Physiology

Song, Sihong
Alpha-1 antitrypsin protects beta cells from apoptosis
Diabetes

Phase 1 trial of intramuscular injection of a recombinant adeno-associated virus serotype 2 alpha-1 antitrypsin (AAT) vvector in AAT-deficient adults
Human Gene Therapy

Alpha-1 antitrypsin gene therapy modulates cellular immunity and efficiently prevents type 1 diabetes in nonobese diabetic mice
Human Gene Therapy

Therapeutic level of functional human alpha-1 antitrypsin (hAAT) secreted from murine muscle transduced by adeno-associated virus (rAAV1) vector
Journal of Gene Medicine

A novel antiapoptotic role for alpha-1 antitrypsin in the prevention of pulmonary emphysema
American Journal of Respiratory and Critical Care Medicine

Efficient hepatic delivery and expression from a recombinant adeno-associated virus 8 pseudotyped alpha-1 antitrypsin vector
Molecular Therapy

Enhancing rAAV vector expression in the lung
Journal of Gene Medicine

Intramuscular administration of recombinant adeno-associated virus 2 alpha-1 antitrypsin (rAAV-SERPINA1) vectors in a nonhuman primate model: safety and immunologic aspects
Molecular Therapy

Improved method of recombinant AAV2 delivery for systemic targeted gene therapy
Molecular Therapy

Steffen, Martin
Possible role of the phagocytic protinases, cathepsin B and elastase, in orthotopic liver transplantation
Transplant Proceedings

Stoller, James
Physician alert suggesting Alpha-1 Antitrypsin Deficiency testing in pulmonary function test results
COPD

New findings in PiZZ Alpha-1 Antitrypsin Deficiency related panniculitis. Demonstration of skin polymers and high dosing requirements of intravenous augmentation therapy
Dermatology

Detection of Alpha-1 Antitrypsin Deficiency: a review
Respiratory Medicine

Heritability of lung function in severe Alpha-1 Antitrypsin Deficiency
Human Heredity

Alpha-1 antitrypsin augmentation therapy for PiMZ heterozygotes: a cautionary note
CHEST

Augmentation therapy in Alpha-1 Antitrypsin Deficiency
Expert Opinion on Biological Therapy

Primary care diagnosis of Alpha-1 Antitrypsin Deficiency: issues and opportunities
Cleveland Clinic Journal of Medicine

Risk factors for symptom onset in PiZ Alpha-1 Antitrypsin Deficiency
International Journal of Chronic Obstructive Pulmonary Disease

Therapy for Alpha-1 Antitrypsin Deficiency: pharmacology and clinical recomendations
BioDrugs

IL10 polymorphisms are associated with airflow obstruction in severe Alpha-1 Antitrypsin Deficiency
American Journal of Respiratory Cell and Molecular Biology

Determinants of airflow obstruction in severe Alpha-1 Antitrypsin Deficiency
Thorax

Lung volume reduction surgery in patients with emphysema and Alpha-1 Antitrypsin Deficiency
Annals of Thoracic Surgery

Cost-effectiveness of population screening for Alpha-1 Antitrypsin Deficiency: a decision analysis
COPD

A review of Alpha-1 Antitrypsin Deficiency
COPD

A review of the Alpha-1 Foundation: its formation, impact, and critical success factors
Respiratory Care

Delay in diagnosis of Alpha-1 Antitrypsin Deficiency: a continuing problem
CHEST

Results of a survey of patients with Alpha-1 Antitrypsin Deficiency
Respiration

A review of Alpha-1 Antitrypsin Deficiency
Seminars in Respiratory and Critical Care Medicine

Alpha-1 Antitrypsin Deficiency
Lancet

Mortality in individuals with severe Alpha-1 Antitrypsin Deficiency: findings from the National Heart, Lung, and Blood Institute Registry
CHEST

The bronchopulmonary pathology of Alpha-1 Antitrypsin Deficiency: findings of the Death Review Committee on the national registry for individuals with Severe Deficiency of alpha-1 antitrypsin
Human Pathology

High dose intravenous AAT and plasma neutrophil derived fibrinogen fragments
Thorax

Augmentation therapy for Alpha-1 Antitrypsin Deficiency
Drugs

Alpha-1 Antitrypsin Deficiency: intravenous augmentation therapy: current understanding Thorax Alpha-1 Antitrypsin Deficiency
Thorax

Key current clinical issues in Alpha-1 Antitrypsin Deficiency
Respiratory Care

Alpha-1 Antitrypsin Deficiency: an under-recognized but important issue for respiratory therapists
Respiratory Care

Major components of the direct medical costs of Alpha-1 Antitrypsin Deficiency
CHEST

Augmentation therapy with Alpha-1 Antitrypsin Deficiency: patterns of use and adverse events
CHEST

Asthma features in severe Alpha-1 Antitrypsin Deficiency: experience of the National Heart, Lung, and Blood Institute Registry
CHEST

Cost-effectiveness analysis of augmentation therapy for severe Alpha-1 Antitrypsin Deficiency
American Journal of Respiratory and Critical Care Medicine

Emphysema in nonsmokers: Alpha-1 Antitrypsin Deficiency and other causes
Cleveland Clinic Journal of Medicine

Low levels of nitric oxide and carbon monoxide in Alpha-1 Antitrypsin Deficiency
Journal of Applied Physiology

Biochemical efficacy and safety of a new pooled human plasma alpha-1 antitrypsin, Respitin
CHEST

The direct medical costs of Alpha-1 Antitrypsin Deficiency
CHEST

Formation and current results of a patient-organized registry for Alpha-1 Antitrypsin Deficiency
CHEST

Occupational exposure risks in individuals with PiZ Alpha-1 Antitrypsin Deficiency
American Journal of Respiratory and Critical Care Medicine

Teckman, Jeffrey
Alpha-1 antitrypsin mutant Z protein content in individual hepatocytes correlates with call death in a mouse model
Hepatology

Alpha-1 antitrypsin deficiency in childhood
Seminar in Liver Disease

In vivo post-transcriptional gene silencing of alpha-1 antitrypsin by adeno-associated virus vectors expressing siRNA
Laboratory Investigation

Indomethacin increases liver damage in a murine model of liver injury from alpha-1 antitrypsin deficiency
Hepatology

Alpha-1 Antitrypsin Deficiency: diagnosis, pathophysiology, and management
Current Gastroenterology Reports

Quantitative isolation of alpha-1 antitrypsin mutant Z protein polymers from human an mouse livers and the effect of heat
Hepatology

Lack of effect of oral 4-phenylbutyrate on serum alpha-1 antitrypsin in patients with alpha-1 antitrypsin deficiency: a preliminary study
Journal of Pediatric Gastroenterology and Nutrition

Analyses of hepatocellular proliferation in a mouse model of Alpha-1 Antitrypsin Deficiency
Hepatology

Mitochondrial autophagy and injury in the liver in Alpha-1 Antitrypsin Deficiency
American Journal of Physiology - Gastrointestinal and Liver Physiology

Fasting in alpha-1 antitrypsin deficient liver: constitutive [correction of consultative]
American Journal of Physiology - Gastrointestinal and Liver Physiology

The proteasome participants in degradation of mutant alpha-1 antitrypsin Z in the enoplasmic reticulum of hepatoma-derived hepatocytes
Journal of Biological Chemistry

A naturally occurring nonpolymerogenic mutant of alpha-1 antitrypsin characterized by prolonged retention in the endoplasmic reticulum
Journal of Biological Chemistry

Retention of mutant alpha-1 antitrypsin Z in endoplasmic reticulum is associated with an autophagic response
American Journal of Physiology - Gastrointestinal and Liver Physiology

Role of ubiquitin in proteasomal degradation of mutant alpha-1 antitrypsin Z in the endoplasmic reticulum
American Journal of Physiology - Gastrointestinal and Liver Physiology

Tuder, Rubin
Alpha-1 antitrypsin inhibits caspase-3 activity, preventing lung endothelial cell apoptosis
American Journal of Pathology

A novel antiapoptic role for alpha-1 antitrypsin in the prevention of pulmonary emphysema
American Journal of Respiratory and Critical Care Medicine

Oxidative stress and apoptosis interact and cause emphysema due to vascular endothelial growth factor receptor blockade
American Journal of Respiratory Cell and Molecular Biology

Turino, Gerry
Alpha-1 Antitrypsin Deficieny: Lessons from longetivity
CHEST

Prevalence of Alpha-1 Antitrypsin Deficiency in poorly controlled asthma—Results from the ALA-ACRC low dose theophylline trial
Journal of Asthma

Determinants of airflow obstruction in severe alpha-1-antitrypsin deficiency
Thorax

Aerosolized hyaluronan limits airspace enlargement in a mouse model of cigarette smoke induced pulmonary emphysema
Experimental Lung Research

The detection and quantitation of free desmosine and isodesmosine in human urine and their peptide-bound forms in sputum
Proceedings of the National Academy of Sciences

Asthma features in sever Alpha-1 Antitrypsin Deficiency: experience of the National Heart, Lung, and Blood Institute Registry
CHEST

Feasibility of a clinical trial of augmentation therapy for Alpha-1 Antitrypsin Deficiency. The Alpha-1 Antitrypsin Deficiency Registry Study Group
American Journal of Respiratory and Critical Care Medicine

Vedal, Sverre
Risk factors for symptom onset in PI*Z Alpha-1 Antitrypsin Deficiency
International Journal of Chronic Obstructive Pulmonary Disease

Verfaillie, Catherine
Sequential exposure to cytokines reflecting embryogenesis: the key for in vitro differentiation of adult bone marrow stem cells into functional hepatocyte-like cells
Toxicology Science

Wilson, Andrew
Sustained expression of alpha-1 antitrypsin after transplantation of manipulated hematopoietic stem cells
American Journal of Respiratory Cell and Molecular Biology

Wood, Alice
Mortality in Alpha-1 Antitrypsin Deficiency in the United Kingdom
Respiratory Medicine

Phenotypic differences in alpha-1 antitrypsin-deficient sibling pairs may relate to genetic variation
COPD

Matrix metalloprotease polymorphisms are associated with gas transfer in Alpha-1 Antitrypsin Deficiency
Therapeutic Advances in Respiratory Disease

The TNF alpha gene relates to clinical phenotype in Alpha-1 Antitrypsin Deficiency
Respiratory Research

The genetics of chronic obstructive pulmonary disease
Respiratory Research

Yarborough, Mark
Responsible patient advocacy: perspectives from the Alpha-1 Foundation
American Journal of Medical Genetics Part A

Yu, Hong
Inhibition of mouse hepatocyte apoptosis by anti-caspase-12 small interfering RNA
Chinese Journal of Hepatology

Zern, Mark
Cell transplantation to replace whole liver transplantation
Gastroenterology

Differentiation in enrichment of hepatocyte-like cells from human embryonic stem cells in vitro and in vivo
Stem Cells

Recent progress of pathogenesis and treatment in Alpha-1 Antitrypsin Deficiency
Japanese Journal of Clinical Medicine

Ribozyme-mediated specific gene replacement of the alpha-1 antitrypsin gene in human hepatoma cells
Journal of Hepatology

A novel SV40-based vector successfully transduces and expresses an alpha-1 antitrypsin ribozyme in a human hepatoma-derived cell line
Gene Therapy

Zhou, Aiwu
Targeting a surface cavity of alpha-1 antitrypsin to prevent conformational disease
Journal of Biological Chemistry

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