Section I. The biology of human huntingtin and Huntington's disease 1. Huntington's disease clinical course and imaging 2. Huntington's disease genetics and genetic modifiers 3. Biomarkers for Huntington's disease 4. The neurobiology of huntingtin in the human brain Section II. Molecular and cellular bases of Huntington's disease pathogenesis 5. Proteostasis dysregulation in HD 6. Organelle dysfunction in HD: mitochondria and autophagy impairment. 7. Aberrant RNA-mediated toxic mechanisms in H 8. HD GWAS genes as genetic modifiers of Huntingtin CAG repeat instability in vivo 9. Biochemical mechanisms of CAG repeat instability in HD: opportunities for therapeutic targeting 10. RAN translation in HD 11. Huntingtin protein-protein interactions: from biology to therapeutic targets 12. The glial contribution to HD pathogenesis 13. Pathological neurocircuitry and synaptic toxicities in HD Section III. Systems biology studies of HD pathogenesis 14. Systems biology approaches to understanding HD pathogenesis and validating therapeutic targets in vivo 15 Systems biology studies of HD patient cells: from iPSCs to postmortem brain 16. Unbiased genome-wide approaches to identify vulnerability factors in HD Section IV. The tools and model systems for basic and translational research in HD 17. HD mouse genetic models 18. Large animal models of HD 19. Deep learning and deep phenotyping of HD iPSC cells: applications to study biology and testing therapeutics 20. HD patient-derived organoids: unraveling neurodevelopmental mechanisms in HD 21. Striatal neuronal models of HD through direct-conversion: modeling age-dependent disease phenotypes 22. Innovative viral tools to study HD pathogenic mechanisms and deliver therapeutics
