Description
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 of Contents
1. Huntington's Disease: Clinical Features, Genetic Diagnosis, and Brain ImagingCarlos Estevez-Fraga, Mitsuko Nakajima and Sarah J. Tabrizi2. 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. Gantman3. Huntington's Disease Genetics: Implications for PathogenesisMarcy E. Macdonald, Jong-Min Lee and James F. Gusella4. The Instability of the Huntington's Disease CAG Repeat MutationVanessa C. Wheeler, Joseph C. Stone, Thomas H. Massey and Ricardo Mouro Pinto5. Mechanisms of Somatic CAG -Repeat Expansions in Huntington's DiseaseAmit L. Deshmukh, Terence Gall-Duncan and Christopher E. Pearson6. RNA-Mediated Pathogenic Mechanisms in Huntington's DiseaseGillian P. Bates, Sandra Fienko, Christian Landles and Aikaterini-Smaragdi Papadopoulou7. Huntingtin Protein-protein Interactions: From Biology to Therapeutic TargetsEduardo Silva Ramos, Todd M. Greco, Ileana M. Cristea and Erich E. Wanker8. Repeat-Associated Non-AUG (RAN) Translation and Huntington's Disease: Pathology, Mechanistic and Therapeutic PerspectivesMonica Banez-Coronel, John Douglas Cleary and Laura P.W. Ranum9. Proteostasis Function and Dysfunction in Huntington's DiseaseJuliana Abramovich, Korbin Kleczko, Vincent Masto and Judith Frydman10. Autophagy and Huntington's DiseaseKatherine R. Croce, Hilary Grosso Jasutkar and Ai Yamamoto11. SUMO Modification in Huntington's Disease: Unraveling Complex Mechanisms for Therapeutic InsightsCharlene Smith, Joan S. Steffan and Leslie M. Thompson12. Selective Vulnerability in Huntington's Disease: From Excitotoxicity, MitochondrialDysfunction, and Transcription Dysregulation to Therapeutic OpportunityJacob S. Deyell, Ravinder Gulia and Albert R. La Spada13. Pathophysiology of Synapses and Circuits in Huntington DiseaseMarja D. Sepers, James Mackay and Lynn A. Raymond14. The Role of Glial Pathology in Huntington's DiseaseSteven A. Goldman15. Systems Biology Study of Huntington's DiseaseLeonardo E. Dionisio, Peter Langfelder, Jeffrey S. Aaronson, Jim Rosinski and X. William Yang16. Unbiased Genome-Wide Approaches to Identify Vulnerability Factors in Huntington's DiseaseSuphinya Sathitloetsakun and Myriam Heiman17. Striatal Neuronal Models of Huntington's Disease Via Direct Conversion: Modeling Age-dependent Disease PhenotypesYoung Mi Oh, Seong Won Lee and Andrew S. Yoo18. Genetic Mouse Models to Explore Huntington's Disease Mechanisms and Therapeutic StrategiesMichelle Gray, Scott O. Zeitlin, Aida Moran-Reyna and Jeh-Ping Liu19. Huntington's Disease: From Large Animal Models to HD Gene TherapySen Yan, Xiao-Jiang Li and Shihua Li20. Deep Learning and Deep Phenotyping of HD iPSCs: Applications to Study Biology and Test TherapeuticsSteven Finkbeiner21. The Promise of an Underappreciated Therapeutic Target: Sleep and Circadian Rhythm Dysfunction in Huntington's DiseaseChristopher S. Colwell, Weiyi Tan and A. Jennifer Morton22. Huntingtin Lowering TherapeuticsNeil Aronin, Miguel E. Sena-Esteves, Anastasia Khvorova, Marian Difiglia and Michael Brodsky23. Gene Editing for HD: Therapeutic ProspectsRichard Z. Chen and Thomas F. Vogt24. Current Clinical Trials of New Therapeutic Agents for Huntington's DiseaseBlair R. Leavitt
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