Protein Homeostasis Diseases

<p>I. Introduction of protein folding and homeostasis<br>1. Protein folding: how, why, and beyond <br>2. Protein homeostasis and disease </p> <p>II. Protein folding and homeostasis at the organismal and proteomic scales<br>3. Caenorhabditis elegans as a model organism for protein homeostasis diseases<br>4. Proteome-scale studies of protein stability <br>5. Classifying disease-associated variants using measures of protein activity and stability </p> <p>III. Protein homeostasis disturbance in disease: Genetics, mechanisms, and modulation by natural ligands<br>6. Protein destabilization and degradation as a mechanism for hereditary disease <br>7. Detection of amyloid aggregation in living systems <br>8. Molecular mechanisms of amyloid aggregation in human proteinopathies <br>9. Metals and amyloid gain-of-toxic mechanisms in neurodegenerative diseases <br>10. Vitamin B6-dependent enzymes and disease <br>11. Galactosemia: opportunities for novel therapies <br>12. Protein homeostasis and regulation of intracellular trafficking of G protein-coupled receptors <br>13. Structure-guided discovery of pharmacological chaperones targeting protein conformational and misfolding diseases <br>14. Virtual screening in drug discovery: a precious tool for a still-demanding<br>challenge <br>15. Differential scanning fluorimetry in the screening and validation of pharmacological chaperones for soluble and membrane proteins <br>16. Cellular high-throughput screening <br>17. High-throughput screening for intrinsically disordered proteins by using biophysical methods <br>18. Natural and pharmacological chaperones against accelerated protein degradation: uroporphyrinogen III synthase and congenital erythropoietic porphyria</p>