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Cell Press Reviews: Stem Cells to Model and Treat Disease informs, inspires, and connects stem cell researchers and clinicians at all stages in their careers with timely, comprehen… Read more
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Cell Press Reviews: Stem Cells to Model and Treat Disease informs, inspires, and connects stem cell researchers and clinicians at all stages in their careers with timely, comprehensive reviews written by leaders in the field and curated by Cell Press editors. The publication offers a broad view of some of the most compelling topics in stem cell research including:
Contributions come from leading voices in the field, including:
- Shinya Yamanaka, Recipient of the 2012 Nobel Prize for Physiology or Medicine, Recipient of the 2012 Millennium Technology Prize, Professor and Director of the Center for iPS Cell Research and Application at Kyoto University, Senior Investigator at the Gladstone Institute of Cardiovascular Disease, L.K. Whittier Foundation Investigator in Stem Cell Biology and Professor at the University of California, San Francisco
- George Q. Daley, Samuel E. Lux IV Professor of Hematology/Oncology at Harvard Medical School and Director for the Stem Cell Transplantation Program at Boston Children’s Hospital
- Irving Weissman, Member of National Academy of Sciences, Virgina & D.K Ludwig Professor for Clinical Investigation in Cancer Research, and Director for Institute of Stem Cell Biology and Regenerative Medicine at Stanford University of Medicine
Cell Press Reviews: Stem Cells to Model and Treat Disease is part of the Cell Press Reviews series, which features reviews published in Cell Press primary research and Trends reviews journals.
About Cell Press
Contributors
Preface
Chapter 1. Stem Cell Therapies Could Change Medicine… If They Get the Chance
Summary
Introduction
Doing Harm
The Therapeutic Entity is the Stem Cell Itself
In Vivo Veritas
An Unexpected but Potent Barrier: Business Development
In Closing....
References
Chapter 2. Why Is It Taking So Long to Develop Clinically Competitive Stem Cell Therapies for CNS Disorders?
Summary
Introduction
The Problem of Generating the Right Cells and Understanding Their Mechanisms of Action
The Problem of Using the Right Animal Model and Behavioral Tests
The Problem of Distribution and Progression of Pathology
The Problem of Translating Basic Research Findings to Patients
The Problem of Competing Therapeutic Approaches
The Problem of Costs
Conclusions
References
Chapter 3. Assessing the Safety of Stem Cell Therapeutics
Acknowledgments
Summary
Introduction
Current Status of Stem Cell Therapeutics and the Safety Challenge
The Safety Issues: Preclinical Assessment
The Safety Issues: Clinical Assessment
Current Assays for Stem Cell Therapy Safety Assessment
Regulation of Stem Cell Therapeutics
Proposals
References
Chapter 4. The Promise and Perils of Stem Cell Therapeutics
Acknowledgments
Summary
Cell Therapeutics: The Current Standard of Care
Lessons from the Historical Development of HSC Transplantation
Stem Cell Therapeutics: Frontline Clinical Trials and Medical Innovations
Conclusions
References
Chapter 5. Reprogramming Cellular Identity for Regenerative Medicine
Acknowledgments
Summary
Introduction
Manipulating Cellular Identity In Vitro
Reprogramming-Based Disease Models Provide a New Platform for Disease Research
Reprogramming Imperfectly Resets the Epigenome to an ES Cell-Like State
Sources of Epigenetic Variation In Vivo: Environment and Stochasticity
Genetic, Environmental, and Epigenetic Contributions to Complex Diseases
Value of Stem Cell-Based Disease Modeling
Conclusions
References
Chapter 6. Cardiac Stem Cell Therapy and the Promise of Heart Regeneration
Acknowledgments
Summary
Introduction
Established Principles
Unresolved Questions
Conclusions
References
Chapter 7. Next-Generation Regenerative Medicine: Organogenesis from Stem Cells in 3D Culture
Summary
Introduction: Key Roles for Noncentralized Patterning Mechanisms in Organogenesis
Self-Organization of Neuroectodermal Structures
Endodermal Tissues
Control of Epithelial-Mesenchymal Interactions
Perspectives for Future Medical Applications
References
Chapter 8. Induced Pluripotent Stem Cells: Past, Present, and Future
Acknowledgments
Summary
Introduction
The Merging of Three Scientific Streams Led to the Production of iPSCs
Maturation and Understanding of iPSC Technology
New Scientific Streams have Emerged from iPSC Technology
The Big Question: are iPSCs Different from ESCs?
Is there a “Dark Side” to Induced Pluripotency?
Why are ESCs and iPSCs So Remarkably Similar?
Concluding Thoughts
References
Chapter 9. Remodeling Neurodegeneration: Somatic Cell Reprogramming-Based Models of Adult Neurological Disorders
Acknowledgments
Summary
Introduction
Cell-Fate Plasticity and iPSC Reprogramming
Directed Reprogramming: A Shortcut
Cell Reprogramming-Based Models of Adult Neurological Disorders
AD: Cell-Type-Specific In Vitro Correlates of Human Brain Pathology
PD and the Role of Environmental Stressors
ALS and Nonautonomous Mechanisms of Disease
Therapeutics: the Endgame
Epigenetic Reprogramming and the Etiology of Neurological Disorders: Nature Versus Nurture
Conclusion
References
Chapter 10. Therapeutic Translation of iPSCs for Treating Neurological Disease
Summary
Introduction
Modeling Neurological and Psychiatric Diseases In Vitro with Pluripotent Stem Cells
Translational and Clinical Opportunities for Pluripotent Stem Cells
Conclusion/Future Directions
References
Chapter 11. Modeling Human Disease with Pluripotent Stem Cells: From Genome Association to Function
Acknowledgments
Summary
Resolving the Taxonomy of Human Disease
hPSCs as a Powerful Tool for Modeling Human Disease
Challenges and Approaches to Disease Modeling with hPSCs
Illuminating Disease Mechanisms with hPSC-Based Disease-Modeling Data
Identification of Shared Molecular Disease Mechanisms between Disease Subtypes
hPSCs as a Tool to Sort and Curate Genomics Data
Modeling Non-Cell-Autonomous Contributions to Human Disease with hPSCs
Concluding Remarks
References
Chapter 12. How Can Human Pluripotent Stem Cells Help Decipher and Cure Huntington’s Disease?
Summary
Introduction
PSC-Based Therapy for HD
Human PSCs for Gene Therapy Validations
Exploring Molecular Mechanisms of HD Using Human PSC Lines
So, What can PSC Neural Derivatives Tell Us about HD?
Using Human PSC Lines for Drug Discovery in HD
Conclusion
References
Chapter 13. Integrating Human Pluripotent Stem Cells into Drug Development
Acknowledgments
Summary
Introduction
Shifting Paradigms in Drug Discovery
Building More Physiologically Relevant In Vitro Assays
IVD hPSC-Derived Cells for Drug Discovery
Challenges to Implementing IVD hPSC-Derived Cell Models in Drug Discovery
Introduction of New Technology
Licensing, Intellectual Property, and Legal Issues
Cell-Based Issues
Assay-Based Issues
Future Use of IVD hPSC-Derived Cells in Drug Discovery
References
Chapter 14. Process Engineering of Human Pluripotent Stem Cells for Clinical Application
Acknowledgments
Summary
The Potential for hPSC-Based Therapies
Transferring hPSCs to the Clinic: Crucial Needs in Process Engineering
Process Engineering of hPSCs
Final Remarks and Future Perspectives
References
Chapter 15. Mesenchymal Stem Cells: Therapeutic Outlook for Stroke
Summary
Current Therapies for Stroke
Bone Marrow-Derived Mesenchymal Stem Cells (MSCs)
MSC Transplantation in Experimental Stroke Models
Clinical Studies on Intravenously Delivered Human MSCs
Concluding Remarks and Future Prospects
References
Chapter 16. The Potential of Stem Cells as an In Vitro Source of Red Blood Cells for Transfusion
Summary
Unmet Transfusion Needs
The Search for the Optimal Stem Cell Source
Economic and Logistical Challenges
Intermediate Therapeutic Goals
Safety Concerns
Concluding Remarks
Web Resources
References
Chapter 17. Hematopoietic-Stem-Cell-Based Gene Therapy for HIV Disease
Acknowledgments
Summary
Background
Identification and Expansion of HSCS
Introduction of De Novo Gene Functions into HSCs
Selection of Gene Functions to Confer Resistance to HIV
In Vivo Engraftment of Transduced HSCs
Pretransplant Conditioning Regimens
Design of HSC-Based Gene Therapy Clinical Trials in Humans
Concluding Remarks
References
Chapter 18. Stem Cells in Translation
iPSCs in Clinics
Transplantation
Brain in a Dish
Cardiac Transdifferentiation
Stem Cell and Diabetes
Cancer Stem Cells
Secrets in the Egg
Power of Tissue Culture
Challenges Ahead
Index