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January 24, 2006; 66 (1 suppl 1) Articles

Developing therapeutics for the diseases of protein misfolding

Barnaby C.H. May, Cedric Govaerts, Fred E. Cohen
First published December 16, 2005, DOI: https://doi.org/10.1212/01.wnl.0000192309.26571.a7
Barnaby C.H. May
PhD
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Cedric Govaerts
PhD
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Fred E. Cohen
MD, PhD
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Developing therapeutics for the diseases of protein misfolding
Barnaby C.H. May, Cedric Govaerts, Fred E. Cohen
Neurology Jan 2006, 66 (1 suppl 1) S118-S122; DOI: 10.1212/01.wnl.0000192309.26571.a7

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Abstract

Our current structural and biologic understanding of the misfolding diseases has restricted the development of therapies that target these diseases at a molecular level. The prion diseases are illustrative of this group of misfolding disorders and provide a model system for therapeutic intervention. Strategies to inhibit the replication and accumulation of the prion protein are being developed and have entered animal and clinical studies. Due to the underlying molecular basis of this disease class, many of the therapeutic approaches used to target prion misfolding have parallels in other misfolding diseases.

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  • Article
    • Abstract
    • Structural studies of the prion protein may provide a structural basis for drug design.
    • Drug discovery targeting the misfolding diseases: Common strategies in the face of common problems.
    • Screening for compounds that disrupt misfolding of the prion protein.
    • Structure-based drug design of dominant-negative prion mimetics.
    • Perspective.
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