Alpha 1-Antitrypsin Deficiency — Epigenetic Regulation of Immunity in Alpha-1 Anti-trypsin Deficiency
Citation(s)
Akimova T, Beier UH, Liu Y, Wang L, Hancock WW Histone/protein deacetylases and T-cell immune responses. Blood. 2012 Mar 15;119(11):2443-51. doi: 10.1182/blood-2011-10-292003. Epub 2012 Jan 12. Review.
Bouchecareilh M, Balch WE Proteostasis, an emerging therapeutic paradigm for managing inflammatory airway stress disease. Curr Mol Med. 2012 Aug;12(7):815-26. Review.
Bouchecareilh M, Hutt DM, Szajner P, Flotte TR, Balch WE Histone deacetylase inhibitor (HDACi) suberoylanilide hydroxamic acid (SAHA)-mediated correction of a1-antitrypsin deficiency. J Biol Chem. 2012 Nov 2;287(45):38265-78. doi: 10.1074/jbc.M112.404707. Epub 2012 Sep 20.
Breton CV, Byun HM, Wenten M, Pan F, Yang A, Gilliland FD Prenatal tobacco smoke exposure affects global and gene-specific DNA methylation. Am J Respir Crit Care Med. 2009 Sep 1;180(5):462-7. doi: 10.1164/rccm.200901-0135OC. Epub 2009 Jun 4.
Miller RL, Ho SM Environmental epigenetics and asthma: current concepts and call for studies. Am J Respir Crit Care Med. 2008 Mar 15;177(6):567-73. doi: 10.1164/rccm.200710-1511PP. Epub 2008 Jan 10. Review.
Natoli G, Ghisletti S, Barozzi I The genomic landscapes of inflammation. Genes Dev. 2011 Jan 15;25(2):101-6. doi: 10.1101/gad.2018811. Review.
Natoli G, Testa G, De Santa F The future therapeutic potential of histone demethylases: A critical analysis. Curr Opin Drug Discov Devel. 2009 Sep;12(5):607-15. Review.
Seersholm N, Kok-Jensen A, Dirksen A Survival of patients with severe alpha 1-antitrypsin deficiency with special reference to non-index cases. Thorax. 1994 Jul;49(7):695-8. Erratum in: Thorax 1994 Nov;49(11):1184. Thorax 1998 Jan;53(1):78.
Seersholm N, Wencker M, Banik N, Viskum K, Dirksen A, Kok-Jensen A, Konietzko N Does alpha1-antitrypsin augmentation therapy slow the annual decline in FEV1 in patients with severe hereditary alpha1-antitrypsin deficiency? Wissenschaftliche Arbeitsgemeinschaft zur Therapie von Lungenerkrankungen (WATL) alpha1-AT study group. Eur Respir J. 1997 Oct;10(10):2260-3.
Silverman EK, Sandhaus RA Clinical practice. Alpha1-antitrypsin deficiency. N Engl J Med. 2009 Jun 25;360(26):2749-57. doi: 10.1056/NEJMcp0900449. Review.
Stockley RA, Bayley DL, Unsal I, Dowson LJ The effect of augmentation therapy on bronchial inflammation in alpha1-antitrypsin deficiency. Am J Respir Crit Care Med. 2002 Jun 1;165(11):1494-8.
Szyf M Epigenetics, DNA methylation, and chromatin modifying drugs. Annu Rev Pharmacol Toxicol. 2009;49:243-63. doi: 10.1146/annurev-pharmtox-061008-103102. Review.
Teckman JH, Jain A Advances in alpha-1-antitrypsin deficiency liver disease. Curr Gastroenterol Rep. 2014 Jan;16(1):367. doi: 10.1007/s11894-013-0367-8. Review.
Wolff GL, Kodell RL, Moore SR, Cooney CA Maternal epigenetics and methyl supplements affect agouti gene expression in Avy/a mice. FASEB J. 1998 Aug;12(11):949-57.
Yang IV, Schwartz DA Epigenetic control of gene expression in the lung. Am J Respir Crit Care Med. 2011 May 15;183(10):1295-301. doi: 10.1164/rccm.201010-1579PP. Review.
Defining Epigenetic Regulation of Immunity in Alpha-1 Anti-trypsin Deficiency
Interventional studies are often prospective and are specifically tailored to evaluate direct impacts of treatment or preventive measures on disease.
Observational studies are often retrospective and are used to assess potential causation in exposure-outcome relationships and therefore influence preventive methods.
Expanded access is a means by which manufacturers make investigational new drugs available, under certain circumstances, to treat a patient(s) with a serious disease or condition who cannot participate in a controlled clinical trial.
Clinical trials are conducted in a series of steps, called phases - each phase is designed to answer a separate research question.
Phase 1: Researchers test a new drug or treatment in a small group of people for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.
Phase 2: The drug or treatment is given to a larger group of people to see if it is effective and to further evaluate its safety.
Phase 3: The drug or treatment is given to large groups of people to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.
Phase 4: Studies are done after the drug or treatment has been marketed to gather information on the drug's effect in various populations and any side effects associated with long-term use.