Huntingtons Disease: Pathogenic Mechanisms and Implications for Therapeutics

Huntington's disease (HD) is one of the most common dominantly inherited neurodegenerative disorders, characterized by a clinical triad 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 thinking on therapeutic strategies for HD. Chapters explore areas that include the clinical features and genetic studies of HD, the cellular and molecular biology of normal huntingtin, a range of HD models, the diverse pathogenic mechanisms linked to mutant huntingtin, new approaches to HD pathogenesis, as well as emerging preclinical therapeutic approaches and clinical programs in the field. Reviews the clinical course and genetics of HDReviews the biology of human huntingtin and HD-relevant cell typesReviews the wide range of pathobiology associated with mutant huntingtinReviews genetic studies of HD and how these studies are informing the development of new therapeutic approachesReviews 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 approachesProvides an overview of new therapeutic approaches and current clinical programs in HD INDICE: 1. Huntington’s Disease: Clinical Features, Genetic Diagnosis, and Brain Imaging Carlos Estevez-Fraga, Mitsuko Nakajima and Sarah J. Tabrizi 2. Revolutionizing Clinical Research and Communication in Huntington’s Disease: The Huntington’s Disease Integrated Staging System (HD-ISS) Cristina Sampaio, Jeffrey D. Long, Alexandra Mansbach, Sarah J. Tabrizi, Emily C. Gantman 3. Huntington’s Disease Genetics: Implications for Pathogenesis Marcy E. Macdonald, Jong-Min Lee and James F. Gusella 4. The Instability of the Huntington’s Disease CAG Repeat Mutation Vanessa C. Wheeler, Joseph C. Stone, Thomas H. Massey and Ricardo Mouro Pinto 5. Mechanisms of Somatic CAG -Repeat Expansions in Huntington’s Disease Amit L. Deshmukh, Terence Gall-Duncan and Christopher E. Pearson 6. RNA-Mediated Pathogenic Mechanisms in Huntington’s Disease Gillian P. Bates, Sandra Fienko, Christian Landles and Aikaterini-Smaragdi Papadopoulou 7. Huntingtin Protein-protein Interactions: From Biology to Therapeutic Targets Eduardo Silva Ramos, Todd M. Greco, Ileana M. Cristea and Erich E. Wanker 8. Repeat-Associated Non-AUG (RAN) Translation and Huntington’s Disease: Pathology, Mechanistic and Therapeutic Perspectives Monica Banez-Coronel, John Douglas Cleary and Laura P.W. Ranum 9. Proteostasis Function and Dysfunction in Huntington’s Disease Juliana Abramovich, Korbin Kleczko, Vincent Masto and Judith Frydman 10. Autophagy and Huntington’s Disease Katherine R. Croce, Hilary Grosso Jasutkar and Ai Yamamoto 11. SUMO Modification in Huntington’s Disease: Unraveling Complex Mechanisms for Therapeutic Insights Charlene Smith, Joan S. Steffan and Leslie M. Thompson 12. Selective Vulnerability in Huntington’s Disease: From Excitotoxicity, Mitochondrial Dysfunction, and Transcription Dysregulation to Therapeutic Opportunity Jacob S. Deyell, Ravinder Gulia and Albert R. La Spada 13. Pathophysiology of Synapses and Circuits in Huntington Disease Marja D. Sepers, James Mackay and Lynn A. Raymond 14. The Role of Glial Pathology in Huntington’s Disease Steven A. Goldman 15. Systems Biology Study of Huntington’s Disease Leonardo E. Dionisio, Peter Langfelder, Jeffrey S. Aaronson, Jim Rosinski and X. William Yang 16. Unbiased Genome-Wide Approaches to Identify Vulnerability Factors in Huntington’s Disease Suphinya Sathitloetsakun and Myriam Heiman 17. Striatal Neuronal Models of Huntington’s Disease Via Direct Conversion: Modeling Age-dependent Disease Phenotypes Young Mi Oh, Seong Won Lee and Andrew S. Yoo 18. Genetic Mouse Models to Explore Huntington’s Disease Mechanisms and Therapeutic Strategies Michelle Gray, Scott O. Zeitlin, Aida Moran-Reyna and Jeh-Ping Liu 19. Huntington’s Disease: From Large Animal Models to HD Gene Therapy Sen Yan, Xiao-Jiang Li and Shihua Li 20. Deep Learning and Deep Phenotyping of HD iPSCs: Applications to Study Biology and Test Therapeutics Steven Finkbeiner 21. The Promise of an Underappreciated Therapeutic Target: Sleep and Circadian Rhythm Dysfunction in Huntington’s Disease Christopher S. Colwell, Weiyi Tan and A. Jennifer Morton 22. Huntingtin Lowering Therapeutics Neil Aronin, Miguel E. Sena-Esteves, Anastasia Khvorova, Marian Difiglia and Michael Brodsky 23. Gene Editing for HD: Therapeutic Prospects Richard Z. Chen and Thomas F. Vogt 24. Current Clinical Trials of New Therapeutic Agents for Huntington’s Disease Blair R. Leavitt

• ISBN: 978-0-323-95672-7
• Editorial: Academic Press
• Encuadernacion: Rústica
• Páginas: 618
• Fecha Publicación: 22/02/2024
• Nº Volúmenes: 1
• Idioma: Inglés