ArticleBiologics : targets & therapy2009
Gene targeted therapeutics for liver disease in alpha-1 antitrypsin deficiency.
Article in Biologics : targets & therapy, 2009. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 9 papers, 1 of them a synthesis that pooled it.
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Who cites it
9 citing papers in PubMed, 1 synthesis or guideline pooled it, 22 citations in OpenAlex.
- Alpha1-Antitrypsin Deficiency: Transition of Care for the Child With AAT Deficiency into Adulthood.Current pediatric reviews · 2019Pooled it
- Decrease in alpha-1 antiproteinase antitrypsin is observed in primary Sjogren's syndrome condition.Autoimmunity · 2020Article
- SERPINA1 mRNA as a Treatment for Alpha-1 Antitrypsin Deficiency.Journal of nucleic acids · 2018Article
- Rapid renal alpha-1 antitrypsin gene induction in experimental and clinical acute kidney injury.PloS one · 2014Article
- Identification of protease inhibitors by a fast fluorimetric assay.Molecular biotechnology · 2013Article
- 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 molecular medicine · 2013Article
- State-of-the-art gene-based therapies: the road ahead.Nature reviews. Genetics · 2011Review
- Z α-1 antitrypsin deficiency and the endoplasmic reticulum stress response.World journal of gastrointestinal pharmacology and therapeutics · 2010Article
- Safety and efficacy of alpha-1-antitrypsin augmentation therapy in the treatment of patients with alpha-1-antitrypsin deficiency.Biologics : targets & therapy · 2009Article
Corrections and comments
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Authors and funding
3 authors at 2 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Alpha-1 antitrypsin (A1AT) is a 52 kDa serine protease inhibitor that is synthesized in and secreted from the liver. Although it is present in all tissues in the body the present consensus is that its main role is to inhibit neutrophil elastase in the lung. A1AT deficiency occurs due to mutations of the A1AT gene that reduce serum A1AT levels to <35% of normal. The most clinically significant form of A1AT deficiency is caused by the Z mutation (Glu342Lys). ZA1AT polymerizes in the endoplasmic reticulum of liver cells and the resulting accumulation of the mutant protein can lead to liver disease, while the reduction in circulating A1AT can result in lung disease including early onset emphysema. There is currently no available treatment for the liver disease other than transplantation and therapies for the lung manifestations of the disease remain limited. Gene therapy is an evolving field which may be of use as a treatment for A1AT deficiency. As the liver disease associated with A1AT deficiency may represent a gain of function possible gene therapies for this condition include the use of ribozymes, peptide nucleic acids (PNAs) and RNA interference (RNAi), which by decreasing the amount of aberrant protein in cells may impact on the pathogenesis of the condition.
Indexed as
Identifiers
19707397PMC2726058W2155124722What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.