Alnylam begins ALN-AAT’s Phase I / II trial for alpha-1 liver disease

Alnylam Pharmaceuticals has started the Phase I / II clinical trial with ALN-AAT, a subcutaneously administered investigational RNAi therapeutic targeting alpha-1 antitrypsin (AAT) to treat AAT deficiency-associated liver disease (alpha-1 liver disea…
Source: Drug Development Technology - Category: Pharmaceuticals Source Type: news

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CONCLUSION: Our findings demonstrate that miR-320c inhibits SERPINA1 expression in a hepatic cell line and its levels in blood are associated with lung disease in a cohort of patients with different AAT serum levels. These results suggest that miR-320c can play a role in AAT regulation and could be a biomarker of inflammatory processes in pulmonary diseases. PMID: 32439252 [PubMed - as supplied by publisher]
Source: Archivos de Bronconeumologia - Category: Respiratory Medicine Authors: Tags: Arch Bronconeumol Source Type: research
Homozygosity for the Pi*Z variant of the gene that encodes the alpha-1 antitrypsin peptide (AAT), called the Pi*ZZ genotype, causes a liver and lung disease called alpha-1 antitrypsin deficiency. Heterozygosity (the Pi*MZ genotype) is a risk factor for cirrhosis in individuals with liver disease. Up to 4% of Caucasians have the Pi*MZ genotype; we compared features of adults with and without Pi*MZ genotype among persons without pre-existing liver disease.
Source: Gastroenterology - Category: Gastroenterology Authors: Source Type: research
AbstractThere are a variety of less common diffuse liver diseases that can be asymptomatic or cause severe liver dysfunction. For the majority of them, the association of clinical, laboratory, and imaging findings are needed to narrow the differential diagnosis. In this article, we will review and describe the rarer diffuse liver diseases including drug-related liver disease, inflammatory and infectious diseases, and deposition disorders such as amyloidosis, glycogen storage disease, Wilson ’s disease, and alpha-1 antitrypsin deficiency. Abdominal radiologists should be familiar with the imaging features of different...
Source: Abdominal Imaging - Category: Radiology Source Type: research
α1-antitrypsin deficiency (AATD) is a hereditary disorder associated with a risk of developing liver disease and pulmonary emphysema, and other chronic respiratory disorders (mainly asthma and bronchiectasis); Z variant is the commonest deficient variant of AAT. Determining AAT concentration in serum or plasma and identifying allelic variants by phenotyping or genotyping are fundamental in the diagnosis of AATD. Initial evaluation and annual follow-up measurement of lung function, including post-bronchodilator forced expiratory volume in 1 s and gas transfer inform on disease progression. Lung densitometry is th...
Source: European Respiratory Review - Category: Respiratory Medicine Authors: Tags: COPD and smoking, Genetics Review Source Type: research
CONCLUSIONS: Beside the role in the first diagnostic step of liver injuries, the utility of liver transaminases is also maintained during the follow-up of liver diseases and in their prognostic assessment. PMID: 31994373 [PubMed - as supplied by publisher]
Source: Minerva Gastroenterologica e Dietologica - Category: Gastroenterology Tags: Minerva Gastroenterol Dietol Source Type: research
ConclusionsLiver organoid model recapitulates the key features of Z-AAT deficiency and provides a useful tool for disease modeling.
Source: Hepatology International - Category: Infectious Diseases Source Type: research
In this study, we developed a diagnostic model combining ultrasonography with biomarkers to identify mild NAFLD, with MRS as the reference standard. A total of 422 eligible subjects were enrolled. The serum levels of fibroblast growth factor 21 (FGF21), cytokeratin 18 M65ED, proteinase 3, neutrophil elastase, alpha-1 antitrypsin, and neutrophil elastase/alpha-1 antitrypsin were measured using ELISA assays. We found that among the six biomarkers, only serum FGF21 was independently associated with intrahepatic triglyceride content (IHTC, standardized β = 0.185, P 
Source: Acta Pharmacologica Sinica - Category: Drugs & Pharmacology Authors: Tags: Acta Pharmacol Sin Source Type: research
Conclusions: Generation of liver organoids might be a useful tool to investigate the factors contributing to hepatic disease in AAT deficiency.
Source: European Respiratory Journal - Category: Respiratory Medicine Authors: Tags: Molecular pathology and funct. genomics Source Type: research
Conclusions: To the best of our knowledge, this is the first controlled study to assess the relationship between AATD and ascending aortic diameter, and suggests AATD is associated with accelerated aortic wall degeneration.
Source: European Respiratory Journal - Category: Respiratory Medicine Authors: Tags: Genes and environment Source Type: research
Conclusions: We have identified a genetic signature that differentiates the different risk groups and the presence of emphysema and hepatopathy in AATD patients.
Source: European Respiratory Journal - Category: Respiratory Medicine Authors: Tags: Molecular pathology and funct. genomics Source Type: research
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