Huntington’s Disease: Pathogenic Mechanisms and Implications for Therapeutics
Editat de X. William Yang, Myriam Heiman, Leslie M. Thompsonen Limba Engleză Paperback – 22 feb 2024
- 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
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Specificații
ISBN-13: 9780323956727
ISBN-10: 0323956726
Pagini: 618
Dimensiuni: 191 x 235 mm
Editura: ELSEVIER SCIENCE
ISBN-10: 0323956726
Pagini: 618
Dimensiuni: 191 x 235 mm
Editura: ELSEVIER SCIENCE
Cuprins
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
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
Recenzii
"Written by prominent leaders in the field, this is not only a must-read for clinicians and researchers of Huntington's disease, but also a highly valuable reference for people in neurodegenerative diseases field in general. The authors did an amazing job, with breadth and depth, in covering a wide range of critical aspects including genetics, molecular and cellular mechanisms of pathogenesis, pathology, patient symptoms and animal model phenotypes, and clinical trials. The literatures covered in this book include historical milestone discoveries as well as the most updated advances in research and therapeutic development. It is truly an indispensable resource for us." -- Chenjian Li, Research Fellow, Hoover Institution, Stanford University