Therapeutic approaches to protein-misfolding diseases

FE Cohen, JW Kelly - Nature, 2003 - nature.com
FE Cohen, JW Kelly
Nature, 2003nature.com
Several sporadic and genetic diseases are caused by protein misfolding. These include
cystic fibrosis and other devastating diseases of childhood as well as Alzheimer's,
Parkinson's and other debilitating maladies of the elderly. A unified view of the molecular
and cellular pathogenesis of these conditions has led to the search for chemical chaperones
that can slow, arrest or revert disease progression. Molecules are now emerging that link our
biophysical insights with our therapeutic aspirations.
Abstract
Several sporadic and genetic diseases are caused by protein misfolding. These include cystic fibrosis and other devastating diseases of childhood as well as Alzheimer's, Parkinson's and other debilitating maladies of the elderly. A unified view of the molecular and cellular pathogenesis of these conditions has led to the search for chemical chaperones that can slow, arrest or revert disease progression. Molecules are now emerging that link our biophysical insights with our therapeutic aspirations.
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