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Review

Why has it been so difficult to prove the efficacy of alpha-1-antitrypsin replacement therapy? Insights from the study of disease pathogenesis

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Pages 391-405 | Published online: 17 Aug 2011

Keep up to date with the latest research on this topic with citation updates for this article.

Read on this site (8)

Isadora Suárez-Lorenzo, Elisa Hernández-Brito, Lourdes Almeida-Quintana, Cesar García-de Llanos, Nereida González-Quevedo, Teresa Carrillo-Díaz & Carlos Rodríguez-Gallego. (2022) Alpha-1 antitrypsin deficiency hidden in allegedly normal variants. Journal of Asthma 59:7, pages 1372-1375.
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Susana Seixas & Patricia Isabel Marques. (2021) Known Mutations at the Cause of Alpha-1 Antitrypsin Deficiency an Updated Overview of SERPINA1 Variation Spectrum. The Application of Clinical Genetics 14, pages 173-194.
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Kylie B R Belchamber, Eloise M Walker, Robert A Stockley & Elizabeth Sapey. (2020) Monocytes and Macrophages in Alpha-1 Antitrypsin Deficiency. International Journal of Chronic Obstructive Pulmonary Disease 15, pages 3183-3192.
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Robert A. Sandhaus, James Stocks, Farshid N. Rouhani, Mark Brantly & Pnina Strauss. (2014) Biochemical Efficacy and Safety of a New, Ready-to-Use, Liquid Alpha-1-Proteinase Inhibitor, GLASSIA (Alpha1-Proteinase Inhibitor (Human), Intravenous). COPD: Journal of Chronic Obstructive Pulmonary Disease 11:1, pages 17-25.
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Shuling Guo, Sheri L Booten, Andrew Watt, Luis Alvarado, Susan M Freier, Jeffery H Teckman, Michael L McCaleb & Brett P Monia. (2014) Using antisense technology to develop a novel therapy for α-1 antitrypsin deficient (AATD) liver disease and to model AATD lung disease. Rare Diseases 2:1.
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David A. Lomas. (2013) Twenty Years of Polymers: A Personal Perspective on Alpha-1 Antitrypsin Deficiency. COPD: Journal of Chronic Obstructive Pulmonary Disease 10:sup1, pages 17-25.
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Manish Mohanka, Danai Khemasuwan & James K Stoller. (2012) A review of augmentation therapy for alpha-1 antitrypsin deficiency. Expert Opinion on Biological Therapy 12:6, pages 685-700.
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ST Schmid, J Koepke, M Dresel, A Hattesohl, E Frenzel, J Perez, DA Lomas, E Miranda, T Greulich, S Noeske, M Wencker, H Teschler, C Vogelmeier, S Janciauskiene & AR Koczulla. (2012) The effects of weekly augmentation therapy in patients with PiZZ α1-antitrypsin deficiency. International Journal of Chronic Obstructive Pulmonary Disease 7, pages 687-696.
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Articles from other publishers (6)

Emanuele Stirpe & Floriana Bardaro. (2022) Alpha1-antitrypsin deficiency and asthma. Monaldi Archives for Chest Disease.
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Nancy T. Malintan, Steven D. Buckingham, David A. Lomas & David B. Sattelle. (2019) Calcium signalling in mammalian cell lines expressing wild type and mutant human α1-Antitrypsin. Scientific Reports 9:1.
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Timothy J. Craig & Maria Paula Henao. (2018) Advances in managing COPD related to α 1 -antitrypsin deficiency: An under-recognized genetic disorder . Allergy 73:11, pages 2110-2121.
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Dieter Köhler. (2017) Alpha-1-Antitrypsin-Mangel: Ist die Substitution ein Mythos?. Deutsches Ärzteblatt Online.
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Ji-Hyun Lee. 2017. COPD. COPD 35 54 .
Neda Motamedi-ShadAlistair M. JaggerMaximilian LiedtkeSarah V. FaullArjun Scott NandaEnrico SalvadoriJoshua L. WortChristopher W.M. KayNarinder Heyer-ChauhanElena Miranda, Juan Perez, Adriana OrdóñezImran HaqJames A. IrvingDavid A. Lomas. (2016) An antibody that prevents serpin polymerisation acts by inducing a novel allosteric behaviour. Biochemical Journal 473:19, pages 3269-3290.
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