Published in Biologics on July 13, 2009
Gene transfer of master autophagy regulator TFEB results in clearance of toxic protein and correction of hepatic disease in alpha-1-anti-trypsin deficiency. EMBO Mol Med (2013) 1.22
Safety and efficacy of alpha-1-antitrypsin augmentation therapy in the treatment of patients with alpha-1-antitrypsin deficiency. Biologics (2009) 0.91
Rapid renal alpha-1 antitrypsin gene induction in experimental and clinical acute kidney injury. PLoS One (2014) 0.86
Z α-1 antitrypsin deficiency and the endoplasmic reticulum stress response. World J Gastrointest Pharmacol Ther (2010) 0.83
Role for a bidentate ribonuclease in the initiation step of RNA interference. Nature (2001) 38.75
An RNA-directed nuclease mediates post-transcriptional gene silencing in Drosophila cells. Nature (2000) 23.17
Functional anatomy of siRNAs for mediating efficient RNAi in Drosophila melanogaster embryo lysate. EMBO J (2001) 12.88
Sequence-selective recognition of DNA by strand displacement with a thymine-substituted polyamide. Science (1991) 12.17
Self-splicing RNA: autoexcision and autocyclization of the ribosomal RNA intervening sequence of Tetrahymena. Cell (1982) 12.12
Introduction of a Chimeric Chalcone Synthase Gene into Petunia Results in Reversible Co-Suppression of Homologous Genes in trans. Plant Cell (1990) 10.56
Hydrodynamics-based transfection in animals by systemic administration of plasmid DNA. Gene Ther (1999) 8.28
The mechanism of Z alpha 1-antitrypsin accumulation in the liver. Nature (1992) 5.71
Purification and properties of ribonuclease III from Escherichia coli. J Biol Chem (1968) 5.48
Cirrhosis associated with alpha-1-antitrypsin deficiency: a previously unrecognized inherited disorder. J Lab Clin Med (1969) 5.40
Gene transfer into humans--immunotherapy of patients with advanced melanoma, using tumor-infiltrating lymphocytes modified by retroviral gene transduction. N Engl J Med (1990) 5.27
Administration of an adenovirus containing the human CFTR cDNA to the respiratory tract of individuals with cystic fibrosis. Nat Genet (1994) 4.40
Liver disease in alpha1-antitrypsin deficiency detected by screening of 200,000 infants. N Engl J Med (1976) 4.39
Chemical chaperones mediate increased secretion of mutant alpha 1-antitrypsin (alpha 1-AT) Z: A potential pharmacological strategy for prevention of liver injury and emphysema in alpha 1-AT deficiency. Proc Natl Acad Sci U S A (2000) 3.17
The serpin superfamily of proteinase inhibitors: structure, function, and regulation. J Biol Chem (1994) 3.07
Sustained secretion of human alpha-1-antitrypsin from murine muscle transduced with adeno-associated virus vectors. Proc Natl Acad Sci U S A (1998) 2.80
Preclinical in vivo evaluation of pseudotyped adeno-associated virus vectors for liver gene therapy. Blood (2003) 2.74
Alpha1-antitrypsin deficiency--a model for conformational diseases. N Engl J Med (2002) 2.11
Worldwide racial and ethnic distribution of alpha1-antitrypsin deficiency: summary of an analysis of published genetic epidemiologic surveys. Chest (2002) 2.10
Antisense-RNA regulation and RNA interference. Biochim Biophys Acta (2002) 2.09
A lag in intracellular degradation of mutant alpha 1-antitrypsin correlates with the liver disease phenotype in homozygous PiZZ alpha 1-antitrypsin deficiency. Proc Natl Acad Sci U S A (1994) 1.91
Z alpha1-antitrypsin polymerizes in the lung and acts as a neutrophil chemoattractant. Chest (2004) 1.88
Liver injury in alpha1-antitrypsin deficiency: an aggregated protein induces mitochondrial injury. J Clin Invest (2002) 1.84
Retention of mutant alpha(1)-antitrypsin Z in endoplasmic reticulum is associated with an autophagic response. Am J Physiol Gastrointest Liver Physiol (2000) 1.83
Oxidation of either methionine 351 or methionine 358 in alpha 1-antitrypsin causes loss of anti-neutrophil elastase activity. J Biol Chem (2000) 1.82
A phase I study of an adeno-associated virus-CFTR gene vector in adult CF patients with mild lung disease. Hum Gene Ther (1996) 1.80
RNA interference: from gene silencing to gene-specific therapeutics. Pharmacol Ther (2005) 1.79
Accumulation of PiZ alpha 1-antitrypsin causes liver damage in transgenic mice. J Clin Invest (1989) 1.72
A novel antiapoptotic role for alpha1-antitrypsin in the prevention of pulmonary emphysema. Am J Respir Crit Care Med (2006) 1.72
Antisense properties of duplex- and triplex-forming PNAs. Nucleic Acids Res (1996) 1.63
The isolation of a clone for human alpha 1-antitrypsin and the detection of alpha 1-antitrypsin in mRNA from liver and leukocytes. Biochem Biophys Res Commun (1983) 1.62
alpha 1-Antitrypsin: molecular pathology, leukocytes, and tissue damage. J Clin Invest (1986) 1.61
Kinetics for hybridization of peptide nucleic acids (PNA) with DNA and RNA studied with the BIAcore technique. Biochemistry (1997) 1.59
The alpha 1-antitrypsin gene and its mutations. Clinical consequences and strategies for therapy. Chest (1989) 1.51
Effect of the Z mutation on the physical and inhibitory properties of alpha 1-antitrypsin. Biochemistry (1993) 1.49
The proteasome participates in degradation of mutant alpha 1-antitrypsin Z in the endoplasmic reticulum of hepatoma-derived hepatocytes. J Biol Chem (2001) 1.49
Neutrophil accumulation in the lung in alpha 1-antitrypsin deficiency. Spontaneous release of leukotriene B4 by alveolar macrophages. J Clin Invest (1991) 1.38
Polymers of Z alpha1-antitrypsin co-localize with neutrophils in emphysematous alveoli and are chemotactic in vivo. Am J Pathol (2005) 1.36
Crystal structure of a peptide nucleic acid (PNA) duplex at 1.7 A resolution. Nat Struct Biol (1997) 1.35
Rnai as an experimental and therapeutic tool to study and regulate physiological and disease processes. Annu Rev Physiol (2005) 1.27
Chromosomal localization of the human alpha 1-antitrypsin gene (PI) to 14q31-32. Am J Hum Genet (1985) 1.26
Structural and functional characterization of the abnormal Z alpha 1-antitrypsin isolated from human liver. FEBS Lett (1984) 1.24
Cell-specific expression of alpha 1-antitrypsin in human intestinal epithelium. J Clin Invest (1993) 1.23
Gene therapy: the first two decades and the current state-of-the-art. J Cell Physiol (2007) 1.21
Antitumor activity of small interfering RNA/cationic liposome complex in mouse models of cancer. Clin Cancer Res (2004) 1.19
Alpha-1 antitrypsin Z protein (PiZ) increases hepatic fibrosis in a murine model of cholestasis. Hepatology (2007) 1.15
Sputum chemotactic activity in chronic obstructive pulmonary disease: effect of alpha(1)-antitrypsin deficiency and the role of leukotriene B(4) and interleukin 8. Thorax (2002) 1.11
Effects in live cells of a c-myc anti-gene PNA linked to a nuclear localization signal. Nat Biotechnol (2000) 1.10
Interfering with disease: opportunities and roadblocks to harnessing RNA interference. Trends Mol Med (2003) 1.10
Long-lasting RNAi activity in mammalian neurons. FEBS Lett (2004) 1.08
Yeast mutants deficient in ER-associated degradation of the Z variant of alpha-1-protease inhibitor. Genetics (1996) 1.06
Factors associated with advanced liver disease in adults with alpha1-antitrypsin deficiency. Clin Gastroenterol Hepatol (2005) 1.04
Isolation and properties of recombinant DNA produced variants of human alpha 1-proteinase inhibitor. J Biol Chem (1985) 1.03
In vivo post-transcriptional gene silencing of alpha-1 antitrypsin by adeno-associated virus vectors expressing siRNA. Lab Invest (2007) 1.03
Siiyama (serine 53 (TCC) to phenylalanine 53 (TTC)). A new alpha 1-antitrypsin-deficient variant with mutation on a predicted conserved residue of the serpin backbone. J Biol Chem (1991) 1.01
Does the heterozygous state of alpha-1 antitrypsin deficiency have a role in chronic liver diseases? Interim results of a large case-control study. J Pediatr Gastroenterol Nutr (2006) 1.01
Assignment of human alpha 1-antitrypsin to chromosome 14 by somatic cell hybrid analysis. Proc Natl Acad Sci U S A (1982) 1.01
Indomethacin increases liver damage in a murine model of liver injury from alpha-1-antitrypsin deficiency. Hepatology (2006) 1.00
Heterozygous alpha-I antitrypsin deficiency as a co-factor in the development of chronic liver disease: a review. Neth J Med (2007) 0.98
Alpha1-antitrypsin deficiency: biological answers to clinical questions. Am J Med Sci (2001) 0.97
Dual therapeutic effects of interferon-alpha gene therapy in a rat hepatocellular carcinoma model with liver cirrhosis. Mol Ther (2008) 0.96
Alpha-1 antitrypsin deficiency: a conformational disease associated with lung and liver manifestations. J Inherit Metab Dis (2008) 0.95
Alpha-1-antitrypsin deficiency: outcomes after liver transplantation. Transplant Proc (2008) 0.95
alpha1-Antitrypsin regulates CD14 expression and soluble CD14 levels in human monocytes in vitro. Int J Biochem Cell Biol (2007) 0.94
Late manifestation of chronic liver disease in adults with alpha-1-antitrypsin deficiency. Dig Dis Sci (1987) 0.93
Heterozygous alpha 1-antitrypsin phenotypes in patients with end stage liver disease. Am J Gastroenterol (1997) 0.92
Patterns of haplotype diversity within the serpin gene cluster at 14q32.1: insights into the natural history of the alpha1-antitrypsin polymorphism. Hum Genet (2001) 0.92
Serine protease inhibitors in patients with chronic viral hepatitis. J Hepatol (1997) 0.92
High prevalence of viral infection in adults with homozygous and heterozygous alpha 1-antitrypsin deficiency and chronic liver disease. Ann Intern Med (1992) 0.91
The peptide nucleic acids (PNAs), powerful tools for molecular genetics and cytogenetics. Eur J Hum Genet (2004) 0.91
Cell-specific involvement of HNF-1beta in alpha(1)-antitrypsin gene expression in human respiratory epithelial cells. Am J Physiol Lung Cell Mol Physiol (2002) 0.89
Fat-soluble vitamins and their importance in patients with cholestatic liver diseases. Gastroenterol Clin North Am (1994) 0.88
Liposomes as carriers for DNA-PNA hybrids. J Control Release (2000) 0.88
A naturally occurring nonpolymerogenic mutant of alpha 1-antitrypsin characterized by prolonged retention in the endoplasmic reticulum. J Biol Chem (2001) 0.88
Developing RNase P ribozymes for gene-targeting and antiviral therapy. Cell Microbiol (2004) 0.86
Is heterozygous alpha-1-antitrypsin deficiency type PIZ a risk factor for primary liver carcinoma? Cancer (2000) 0.86
Expression of hereditary hemochromatosis C282Y HFE protein in HEK293 cells activates specific endoplasmic reticulum stress responses. BMC Cell Biol (2007) 0.86
Endoplasmic reticulum stress--a double edged sword for Z alpha-1 antitrypsin deficiency hepatoxicity. Int J Biochem Cell Biol (2008) 0.85
Ribozyme-mediated specific gene replacement of the alpha1-antitrypsin gene in human hepatoma cells. J Hepatol (1999) 0.85
Modification of gene expression and increase in alpha1-antitrypsin (alpha1-AT) secretion after homologous recombination in alpha1-AT-deficient monocytes. Hum Gene Ther (2007) 0.83
Recombinant adeno-associated virus gene therapy for cystic fibrosis and alpha(1)-antitrypsin deficiency. Chest (2002) 0.82
Does alpha1-antitrypsin phenotype PiMZ increase the risk of fibrosis in liver disease due to hepatitis C virus infection? Eur J Gastroenterol Hepatol (2006) 0.81
Small interfering RNA-mediated functional silencing of vasopressin V2 receptors in the mouse kidney. Physiol Genomics (2005) 0.81
Parker B. Francis lectureship. Antitrypsin deficiency, the serpinopathies, and chronic obstructive pulmonary disease. Proc Am Thorac Soc (2006) 0.79
Pathogenesis and pathology of liver disease associated with alpha 1-antitrypsin deficiency. Chest (1996) 0.76
Alpha-1-antitrypsin deficiency resulting in a hitherto unseen presentation of hepatocellular carcinoma: polycythemia but with normal alpha fetoprotein. World J Gastroenterol (2006) 0.76
Low incidence of alpha 1-antitrypsin deficiency among Filipinos with neonatal cholestatis. J Paediatr Child Health (2006) 0.76
[HYPO-ALPHA-1-ANTITRYPSINEMIA]. Verh Dtsch Ges Inn Med (1964) 0.76
Alpha-1 antitrypsin deficiency among Indian children with liver disorders. Indian J Gastroenterol (2006) 0.75
Phase 2 clinical trial of a recombinant adeno-associated viral vector expressing α1-antitrypsin: interim results. Hum Gene Ther (2011) 2.75
Nebulized hypertonic saline decreases IL-8 in sputum of patients with cystic fibrosis. Am J Respir Crit Care Med (2011) 2.37
Clarification of the risk of chronic obstructive pulmonary disease in α1-antitrypsin deficiency PiMZ heterozygotes. Am J Respir Crit Care Med (2014) 2.14
Effect of estrogen on pseudomonas mucoidy and exacerbations in cystic fibrosis. N Engl J Med (2012) 1.93
Z alpha1-antitrypsin polymerizes in the lung and acts as a neutrophil chemoattractant. Chest (2004) 1.88
Anti-proline-glycine-proline or antielastin autoantibodies are not evident in chronic inflammatory lung disease. Am J Respir Crit Care Med (2009) 1.86
TLR-induced inflammation in cystic fibrosis and non-cystic fibrosis airway epithelial cells. J Immunol (2005) 1.67
Secretory leucoprotease inhibitor binds to NF-kappaB binding sites in monocytes and inhibits p65 binding. J Exp Med (2005) 1.65
Endotoxin up-regulates interleukin-18: potential role for gram-negative colonization in sarcoidosis. Am J Respir Crit Care Med (2005) 1.57
Activation of endoplasmic reticulum-specific stress responses associated with the conformational disease Z alpha 1-antitrypsin deficiency. J Immunol (2004) 1.53
Secretory leucoprotease inhibitor prevents lipopolysaccharide-induced IkappaBalpha degradation without affecting phosphorylation or ubiquitination. J Biol Chem (2002) 1.52
Alpha-1 antitrypsin deficiency. Respir Med (2010) 1.51
Granzyme B-dependent proteolysis acts as a switch to enhance the proinflammatory activity of IL-1α. Mol Cell (2011) 1.48
miR-199a-5p silencing regulates the unfolded protein response in chronic obstructive pulmonary disease and α1-antitrypsin deficiency. Am J Respir Crit Care Med (2014) 1.46
miR-31 dysregulation in cystic fibrosis airways contributes to increased pulmonary cathepsin S production. Am J Respir Crit Care Med (2014) 1.45
α1-Antitrypsin activates protein phosphatase 2A to counter lung inflammatory responses. Am J Respir Crit Care Med (2014) 1.45
Adult-onset nemaline myopathy presenting as respiratory failure. Respir Care (2008) 1.40
Neutrophil elastase up-regulates interleukin-8 via toll-like receptor 4. FEBS Lett (2003) 1.38
α-1 Antitrypsin regulates human neutrophil chemotaxis induced by soluble immune complexes and IL-8. J Clin Invest (2010) 1.38
miR-126 is downregulated in cystic fibrosis airway epithelial cells and regulates TOM1 expression. J Immunol (2010) 1.38
Epithelial expression of TLR4 is modulated in COPD and by steroids, salmeterol and cigarette smoke. Respir Res (2007) 1.31
Loss of microbicidal activity and increased formation of biofilm due to decreased lactoferrin activity in patients with cystic fibrosis. J Infect Dis (2004) 1.25
MicroRNAs in inflammatory lung disease--master regulators or target practice? Respir Res (2010) 1.24
Inactivation of human beta-defensins 2 and 3 by elastolytic cathepsins. J Immunol (2003) 1.24
LL-37 complexation with glycosaminoglycans in cystic fibrosis lungs inhibits antimicrobial activity, which can be restored by hypertonic saline. J Immunol (2009) 1.22
Activation of the epidermal growth factor receptor (EGFR) by a novel metalloprotease pathway. J Biol Chem (2008) 1.20
Human Treg responses allow sustained recombinant adeno-associated virus-mediated transgene expression. J Clin Invest (2013) 1.20
Secretory leucoprotease inhibitor impairs Toll-like receptor 2- and 4-mediated responses in monocytic cells. Infect Immun (2004) 1.20
Proteases and antiproteases in chronic neutrophilic lung disease - relevance to drug discovery. Br J Pharmacol (2009) 1.18
Antimicrobial proteins and polypeptides in pulmonary innate defence. Respir Res (2006) 1.18
Elastolytic proteases: inflammation resolution and dysregulation in chronic infective lung disease. Am J Respir Crit Care Med (2005) 1.18
Neutrophil elastase up-regulates cathepsin B and matrix metalloprotease-2 expression. J Immunol (2007) 1.17
Toll-like receptor expression and function in airway epithelial cells. Arch Immunol Ther Exp (Warsz) (2005) 1.09
Sputum Candida albicans presages FEV₁ decline and hospital-treated exacerbations in cystic fibrosis. Chest (2010) 1.09
17Beta-estradiol inhibits IL-8 in cystic fibrosis by up-regulating secretory leucoprotease inhibitor. Am J Respir Crit Care Med (2010) 1.08
Decreased levels of secretory leucoprotease inhibitor in the Pseudomonas-infected cystic fibrosis lung are due to neutrophil elastase degradation. J Immunol (2009) 1.07
Cytosolic pH and the inflammatory microenvironment modulate cell death in human neutrophils after phagocytosis. Blood (2002) 1.06
Evidence for unfolded protein response activation in monocytes from individuals with alpha-1 antitrypsin deficiency. J Immunol (2010) 1.05
Elafin, an elastase-specific inhibitor, is cleaved by its cognate enzyme neutrophil elastase in sputum from individuals with cystic fibrosis. J Biol Chem (2008) 1.03
Selenoprotein S/SEPS1 modifies endoplasmic reticulum stress in Z variant alpha1-antitrypsin deficiency. J Biol Chem (2009) 1.01
Targeting neutrophil elastase in cystic fibrosis. Expert Opin Ther Targets (2008) 1.00
Elafin prevents lipopolysaccharide-induced AP-1 and NF-kappaB activation via an effect on the ubiquitin-proteasome pathway. J Biol Chem (2006) 1.00
The CF-ABLE score: a novel clinical prediction rule for prognosis in patients with cystic fibrosis. Chest (2013) 0.99
Pulmonary proteases in the cystic fibrosis lung induce interleukin 8 expression from bronchial epithelial cells via a heme/meprin/epidermal growth factor receptor/Toll-like receptor pathway. J Biol Chem (2010) 0.98
The circulating proteinase inhibitor α-1 antitrypsin regulates neutrophil degranulation and autoimmunity. Sci Transl Med (2014) 0.97
Tauroursodeoxycholic acid inhibits apoptosis induced by Z alpha-1 antitrypsin via inhibition of Bad. Hepatology (2007) 0.95
Community-acquired pneumonia in older patients: does age influence systemic cytokine levels in community-acquired pneumonia? Respirology (2009) 0.94
Staphylococcus epidermidis polysaccharide intercellular adhesin induces IL-8 expression in human astrocytes via a mechanism involving TLR2. Cell Microbiol (2008) 0.94
The prevalence of alpha-1 antitrypsin deficiency in Ireland. Respir Res (2011) 0.93
Hypertonic saline in treatment of pulmonary disease in cystic fibrosis. ScientificWorldJournal (2012) 0.92
Isolation and identification of cell-specific microRNAs targeting a messenger RNA using a biotinylated anti-sense oligonucleotide capture affinity technique. Nucleic Acids Res (2013) 0.92
The cystic fibrosis neutrophil: a specialized yet potentially defective cell. Arch Immunol Ther Exp (Warsz) (2011) 0.92
Aspergillus-associated airway disease, inflammation, and the innate immune response. Biomed Res Int (2013) 0.91
Airway inflammatory markers in individuals with cystic fibrosis and non-cystic fibrosis bronchiectasis. J Inflamm Res (2013) 0.91
IL-8 dictates glycosaminoglycan binding and stability of IL-18 in cystic fibrosis. J Immunol (2009) 0.91
SLPI and inflammatory lung disease in females. Biochem Soc Trans (2011) 0.91
Alpha-1 antitrypsin: a potent anti-inflammatory and potential novel therapeutic agent. Arch Immunol Ther Exp (Warsz) (2012) 0.91
Regulation of cystic fibrosis transmembrane conductance regulator by microRNA-145, -223, and -494 is altered in ΔF508 cystic fibrosis airway epithelium. J Immunol (2013) 0.90
The effect of Aspergillus fumigatus infection on vitamin D receptor expression in cystic fibrosis. Am J Respir Crit Care Med (2012) 0.89
Dysregulation of TIM-3-galectin-9 pathway in the cystic fibrosis airways. J Immunol (2011) 0.89
Inhibition of Toll-like receptor 2-mediated interleukin-8 production in Cystic Fibrosis airway epithelial cells via the alpha7-nicotinic acetylcholine receptor. Mediators Inflamm (2010) 0.88
Functional study of elafin cleaved by Pseudomonas aeruginosa metalloproteinases. Biol Chem (2010) 0.88
The role and importance of glycosylation of acute phase proteins with focus on alpha-1 antitrypsin in acute and chronic inflammatory conditions. J Proteome Res (2014) 0.88
Secretory leucocyte protease inhibitor inhibits interferon-gamma-induced cathepsin S expression. J Biol Chem (2007) 0.86