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    Therapeutic targeting of misfolding and conformational change in α1-antitrypsin deficiency

    Nyon, Mun Peak and Gooptu, Bibek (2014) Therapeutic targeting of misfolding and conformational change in α1-antitrypsin deficiency. Future Medicinal Chemistry 6 (9), pp. 1047-1065. ISSN 1756-8919.

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    Misfolding and conformational diseases are increasing in prominence and prevalence. Both misfolding and ‘postfolding’ conformational mechanisms can contribute to pathogenesis and can coexist. The different contexts of folding and native state behavior may have implications for the development of therapeutic strategies. α1-antitrypsin deficiency illustrates how these issues can be addressed with therapeutic approaches to rescue folding, ameliorate downstream consequences of aberrant polymerization and/or maintain physiological function. Small-molecule strategies have successfully targeted structural features of the native conformer. Recent developments include the capability to follow solution behavior of α1-antitrypsin in the context of disease mutations and interactions with drug-like compounds. Moreover, preclinical studies in cells and organisms support the potential of manipulating cellular response repertoires to process misfolded and polymer states.


    Item Type: Article
    School: School of Science > Biological Sciences
    Research Centres and Institutes: Structural Molecular Biology, Institute of (ISMB)
    Depositing User: Administrator
    Date Deposited: 30 Jul 2014 17:53
    Last Modified: 06 Dec 2016 10:42


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