Article
[Pharmacological chaperones: a potential therapeutic treatment for conformational diseases].
Medecine sciences : M/S - 1 Jan 2000
Mendre Christiane, Mouillac Bernard
Abstract excerpt
Many genetic and neurodegenerative diseases in humans result from protein misfolding and/or aggregation. These diseases are named conformational diseases. As a result, the misfolded non functional proteins are rejected and misrouted by the cellular quality control system, and cannot play their endogenous physiological roles. Specific compounds (ligands, substrates or inhibitors) known as pharmacological...
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