Neurology
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Published online before print December 16, 2005, doi:10.1212/01.wnl.0000192309.26571.a7)
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Developing therapeutics for the diseases of protein misfolding

Barnaby C.H. May PhD*, Cedric Govaerts PhD, and Fred E. Cohen MD, PhD

From the Institute for Neurodegenerative Diseases (B.C.H.M.) and Department of Cellular and Molecular Pharmacology (C.G., F.E.C.), University of California, San Francisco, CA.


* To whom correspondence should be addressed. E-mail: alchemi{at}itsa.ucsf.edu.

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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