α1-antitrypsin deficiency
Bruce-Hickman, D. and Greene, C.M. and Gooptu, Bibekbrata (2015) α1-antitrypsin deficiency. In: Anzueto, A. and Heijdra, Y. and Hurst, J.R. (eds.) Controversies in COPD. ERS Monographs. European Respiratory Society, pp. 47-84. ISBN 9781849840644.
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Abstract
The emphysema of α1-ATD is the best understood subphenotype of COPD in terms of its genetic basis, disease behaviour and major pathogenic mechanisms. However, this level of insight has not yet translated to a definitive cure, or even a tailored treatment that has proven robustly effective, for α1-ATD. This apparent paradox drives controversy, by implying an urgent need to identify where knowledge is incomplete or existing data are misunderstood. From another perspective, it highlights how subphenotyping is a necessary element but is not sufficient for development of specific treatments to provide the best possible patient-centred care. In this review of ongoing debates in α1-ATD we address, in turn, the involvement of different cell types, the extent of associated clinical phenotypes, the need to identify the optimal role of existing therapeutic options and how to develop new treatments. In doing so, we define important areas that are the subject of further active research, or that in our view require it.
Metadata
Item Type: | Book Section |
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School: | Birkbeck Faculties and Schools > Faculty of Science > School of Natural Sciences |
Depositing User: | Bibekbrata Gooptu |
Date Deposited: | 30 Nov 2015 15:16 |
Last Modified: | 02 Aug 2023 17:17 |
URI: | https://eprints.bbk.ac.uk/id/eprint/12427 |
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