Huntington's Disease

Pathogenic Mechanisms and Implications for Therapeutics

X. William Yang,Myriam Heiman,Leslie M. Thompson

• Éditeur: Academic Press
• Année: 2024
• ISBN:
  9780323956734
• Sujet: Ciencias biológicas
• Sous-sujet: Neurociencia

Huntington's disease (HD) is one of the most common dominantly inherited neurodegenerative disorders, characterized by a clinical trial of movement disorder, cognitive deficits, and psychiatric symptoms. Huntington's Disease: Pathogenic Mechanisms and Implications for Therapeutics, reviews the most up-do-date content on HD pathogenic mechanisms and cutting-edge testing of therapeutic strategies for HD. Chapters explore areas such as, normal huntingtin biology in brain development and function, genetic modifiers of HD in patients, molecular pathogenic mechanism in HD, and mechanisms underlying selective neuronal vulnerability

• Reviews the clinical course and genetics of HD
• Reviews the biology of human huntingtin and HD-relevant cell types
• Reviews the wide range of pathobiology associated with mutant huntingtin
• Reviews genetic studies of HD and how these studies are informing the development of new therapeutic approaches
• Reviews new tools and model systems for basic and translational research in HD, including new human-derived model systems, as well as systems biology and artificial intelligence–driven approaches
• Provides an overview of new therapeutic approaches and current clinical programs in HD

Table des matières

• Cover image
• Title page
• Table of Contents
• Copyright
• Dedication
• Contributors
• Preface
• Chapter 1. Huntington's disease: Clinical features, genetic diagnosis, and brain imaging
• Chapter 2. Revolutionizing clinical research and communication in Huntington's disease: The Huntington's disease integrated staging system (HD-ISS)
• Chapter 3. Huntington's disease genetics: Implications for pathogenesis
• Chapter 4. The instability of the Huntington's disease CAG repeat mutation
• Chapter 5. Mechanisms of somatic CAG-repeat expansions in Huntington's disease
• Chapter 6. RNA-mediated pathogenic mechanisms in Huntington's disease
• Chapter 7. Huntingtin protein–protein interactions: From biology to therapeutic targets
• Chapter 8. Repeat-associated non-AUG (RAN) translation and Huntington's disease: Pathology, mechanistic and therapeutic perspectives
• Chapter 9. Proteostasis function and dysfunction in Huntington's disease
• Chapter 10. Autophagy and Huntington's disease
• Chapter 11. SUMO modification in Huntington's disease: Unraveling complex mechanisms for therapeutic insights
• Chapter 12. Selective vulnerability in Huntington's disease: From excitotoxicity, mitochondrial dysfunction, and transcription dysregulation to therapeutic opportunity
• Chapter 13. Pathophysiology of synapses and circuits in Huntington disease
• Chapter 14. The role of glial pathology in Huntington's disease
• Chapter 15. Systems biology study of Huntington's disease
• Chapter 16. Unbiased genome-wide approaches to identify vulnerability factors in Huntington's disease
• Chapter 17. Striatal neuronal models of Huntington's disease via direct conversion: Modeling age-dependent disease phenotypes
• Chapter 18. Genetic mouse models to explore Huntington's disease mechanisms and therapeutic strategies
• Chapter 19. Huntington's disease: From large animal models to HD gene therapy
• Chapter 20. Deep learning and deep phenotyping of HD iPSCs: Applications to study biology and test therapeutics
• Chapter 21. The promise of an underappreciated therapeutic target: Sleep and circadian rhythm dysfunction in Huntington's disease
• Chapter 22. Huntingtin lowering therapeutics
• Chapter 23. Gene editing for HD: Therapeutic prospects
• Chapter 24. Current clinical trials of new therapeutic agents for Huntington's disease
• Index