The DOI matches its Crossref record; 2 of the 2 references it cites were found in a registry or an index.
Prevention of Transthyretin Amyloid Disease by Changing Protein Misfolding Energetics
Abstract
Genetic evidence suggests that inhibition of amyloid fibril formation by small molecules should be effective against amyloid diseases. Known amyloid inhibitors appear to function by shifting the aggregation equilibrium away from the amyloid state. Here, we describe a series of transthyretin amyloidosis inhibitors that functioned by increasing the kinetic barrier associated with misfolding, preventing amyloidogenesis by stabilizing the native state. The trans-suppressor mutation, threonine 119 → methionine 119, which is known to ameliorate familial amyloid disease, also functioned through kinetic stabilization, implying that this small-molecule strategy should be effective in treating amyloid diseases.
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Walsh, D. M., Klyubin, I., Fadeeva, J. V., Cullen, W. K., Anwyl, R., Wolfe, M. S., Rowan, M. J., & Selkoe, D. J. (2002) · Nature · doi:10.1038/416535a · Cited by 4,291
Inherent toxicity of aggregates implies a common mechanism for protein misfolding diseases
Bucciantini, M., Giannoni, E., Chiti, F., Baroni, F., Formigli, L., Zurdo, J., Taddei, N., Ramponi, G., Dobson, C. M., & Stefani, M. (2002) · Nature · doi:10.1038/416507a · Cited by 2,434
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Balch, W. E., Morimoto, R. I., Dillin, A., & Kelly, J. W. (2008) · Science · doi:10.1126/science.1141448 · Cited by 2,495
Self-citationBiological and Chemical Approaches to Diseases of Proteostasis Deficiency
Powers, E. T., Morimoto, R. I., Dillin, A., Kelly, J. W., & Balch, W. E. (2009) · Annual Review of Biochemistry · doi:10.1146/annurev.biochem.052308.114844 · Cited by 1,149
Self-citationRationalization of the effects of mutations on peptide andprotein aggregation rates
Chiti, F., Stefani, M., Taddei, N., Ramponi, G., & Dobson, C. M. (2003) · Nature · doi:10.1038/nature01891 · Cited by 1,072
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Metadata
- Authors
- Hammarström, P., Wiseman, R. L., Powers, E. T., & Kelly, J. W.
- Venue
- Science
- Published
- 2003-01-31
- Volume
- 299
- Issue
- 5607
- Pages
- 713-716
- ISSN
- 0036-8075 (ISSN-L)
- 1095-9203
- OpenAlex
- W1972373816
- Cited by
- 525
- References
- 2
- FWCI
- 12.76
- Type
- journal article
- Language
- en
- Open access
- closed
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- Hammarström, P., Wiseman, R. L., Powers, E. T., & Kelly, J. W. (2003). Prevention of Transthyretin Amyloid Disease by Changing Protein Misfolding Energetics. Science. https://doi.org/10.1126/science.1079589
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