Developing therapeutics for the diseases of protein misfolding.
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.
May
Barnaby C H
BC
Institute for Neurodegenerative Diseases, University of California, San Francisco, CA 94143-2240, USA. alchemi@itsa.ucsf.edu
Govaerts
Cedric
C
Cohen
Fred E
FE
D000544:Alzheimer Disease / Q000188:drug therapy / Q000378:metabolism; D000690:Amyotrophic Lateral Sclerosis / Q000378:metabolism; D003924:Diabetes Mellitus, Type 2 / Q000378:metabolism; D015195:Drug Design; D006801:Humans; D006816:Huntington Disease / Q000378:metabolism; D007116:Immunization, Passive; D007700:Kinetics; D015394:Molecular Structure; D009154:Mutation; D009419:Nerve Tissue Proteins / Q000737:chemistry / Q000378:metabolism*; D019636:Neurodegenerative Diseases / Q000188:drug therapy* / Q000378:metabolism; D010300:Parkinson Disease / Q000378:metabolism; D018620:PrPSc Proteins / Q000737:chemistry / Q000276:immunology; D017096:Prion Diseases / Q000188:drug therapy / Q000378:metabolism; D011487:Protein Conformation / Q000187:drug effects; D017510:Protein Folding; D017433:Protein Structure, Secondary
D009419:Nerve Tissue Proteins; D018620:PrPSc Proteins