An RNA structure-mediated, posttranscriptional model of human α-1-antitrypsin expression
10.1073/pnas.1706539114 · doi-reference
Alternative transcripts of the SERPINA1 gene in alpha-1 antitrypsin deficiency
10.1186/s12967-015-0585-y · doi-reference
SERPINA1 gene promoter is differentially methylated in peripheral blood mononuclear cells of pregnant women
10.3389/fcell.2020.550543 · doi-reference
SERPINA1 and more? A putative genetic contributor to pulmonary dysfunction in alpha-1 antitrypsin deficiency
10.3390/jcm12051708 · doi-reference
Feasibility of a clinical trial of augmentation therapy for α1-antitrypsin deficiency
10.1164/ajrccm.161.3.9906011 · doi-reference
Two randomised controlled phase 2 studies of the oral neutrophil elastase inhibitor alvelestat in alpha-1 antitrypsin deficiency
10.1183/13993003.01019-2025 · doi-reference
Pharmacokinetics and safety of AZD9668, an oral neutrophil elastase inhibitor, in healthy volunteers and patients with COPD
10.5414/cp201674 · doi-reference
AZD9668: pharmacological characterization of a novel oral inhibitor of neutrophil elastase
10.1124/jpet.111.182139 · doi-reference
Sirtinol inhibits neutrophil elastase activity and attenuates lipopolysaccharide-mediated acute lung injury in mice
10.1038/srep08347 · doi-reference
Single dose escalation studies with inhaled POL6014, a potent novel selective reversible inhibitor of human neutrophil elastase, in healthy volunteers and subjects with cystic fibrosis
10.1016/j.jcf.2019.08.020 · doi-reference
Neutrophil elastase inhibitor (Sivelestat) in the treatment of acute respiratory distress syndrome induced by COVID-19: a multicenter retrospective cohort study
10.1186/s12931-025-03100-4 · doi-reference
Human neutrophil elastase inhibitors: classification, biological-synthetic sources and their relevance in related diseases
10.1111/fcp.12946 · doi-reference
Paucimannose-rich N-glycosylation of spatiotemporally regulated human neutrophil elastase modulates its immune functions
10.1074/mcp.m116.066746 · doi-reference
Mechanisms of emphysema in α1-antitrypsin deficiency: molecular and cellular insights
10.1183/09031936.00096508 · doi-reference
Repopulation of the fibrotic/cirrhotic rat liver by transplanted hepatic stem/progenitor cells and mature hepatocytes
10.1002/hep.26615 · doi-reference
Capturing the conversion of the pathogenic alpha-1-antitrypsin fold by ATF6 enhanced proteostasis
10.1016/j.chembiol.2022.12.004 · doi-reference
Pharmacological activation of ATF6 remodels the proteostasis network to rescue pathogenic GABAA receptors
10.1186/s13578-022-00783-w · doi-reference
Alpha 1-antitrypsin deficiency: a disorder of proteostasis-mediated protein folding and trafficking pathways
10.3390/ijms21041493 · doi-reference
Alpha-1 antitrypsin deficiency: genetics, clinical manifestations, AI prognostics, and advanced imaging in liver disease
10.1097/ms9.0000000000003913 · doi-reference
RNA editing for the treatment of alpha-1 antitrypsin deficiency
10.1093/nar/gkaf1518 · doi-reference
Editing approaches to treat alpha-1 antitrypsin deficiency
10.1016/j.chest.2024.09.038 · doi-reference
Gene therapy for alpha-1 antitrypsin deficiency: an update
10.1080/14712598.2023.2183771 · doi-reference
Adenine base editing reduces misfolded protein accumulation and toxicity in alpha-1 antitrypsin deficient patient iPSC-hepatocytes
10.1016/j.ymthe.2021.06.021 · doi-reference
A compact base editor rescues AATD-associated liver and lung disease in mouse models
10.1016/j.ymthe.2025.08.040 · doi-reference
Long-term therapeutic levels of human alpha-1 antitrypsin in plasma after hydrodynamic injection of nonviral DNA
10.1038/sj.gt.3302065 · doi-reference
Hydrodynamic liver gene transfer mechanism involves transient sinusoidal blood stasis and massive hepatocyte endocytic vesicles
10.1038/sj.gt.3302469 · doi-reference
Asialofetuin liposome-mediated human alpha1-antitrypsin gene transfer in vivo results in stationary long-term gene expression
10.1007/s001090000185 · doi-reference
Human 1-antitrypsin gene transfer to in vivo mouse hepatocytes
10.1089/hum.1996.7.4-531 · doi-reference
Survival advantage of both human hepatocyte xenografts and genome-edited hepatocytes for treatment of α-1 antitrypsin deficiency
10.1016/j.ymthe.2017.09.020 · doi-reference
Amelioration of alpha-1 antitrypsin deficiency diseases with genome editing in transgenic mice
10.1089/hum.2017.227 · doi-reference
Advances in Precision Editing Therapies for Alpha-1 Antitrypsin Deficiency
10.1177/10430342261417356 · doi-reference
Editing out five Serpina1 paralogs to create a mouse model of genetic emphysema
10.1073/pnas.1713689115 · doi-reference
Alpha-1-antitrypsin deficiency: A new paradigm for hepatocellular carcinoma in genetic liver disease
10.1002/hep.20815 · doi-reference
Efficient and targeted transduction of nonhuman primate liver with systemically delivered optimized AAV3B vectors
10.1038/mt.2015.174 · doi-reference
Accumulation of PiZ alpha 1-antitrypsin causes liver damage in transgenic mice
10.1172/jci113999 · doi-reference
Quantitative lipid profiling reveals major differences between liver organoids with normal Pi*M and deficient Pi*Z variants of alpha-1-antitrypsin
10.3390/ijms241512472 · doi-reference
Human iPSC-hepatocyte modeling of alpha-1 antitrypsin heterozygosity reveals metabolic dysregulation and cellular heterogeneity
10.1016/j.celrep.2022.111775 · doi-reference
Liver organoids reproduce alpha-1 antitrypsin deficiency-related liver disease
10.1007/s12072-019-10007-y · doi-reference
Long-term culture of genome-stable bipotent stem cells from adult human liver
10.1016/j.cell.2014.11.050 · doi-reference
Induced pluripotent stem cells model personalized variations in liver disease resulting from α1-antitrypsin deficiency
10.1002/hep.27753 · doi-reference
