Stem Cells in Modeling Human Genetic Diseases PDF Download

Author: Mayana Zatz
Publisher: Springer
ISBN: 3319183141
Category : Science
Languages : en
Pages : 147

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Book Description
While most stem cell books focus on basic aspects and/or cell therapy, this book emphasizes the relevance of stem cells obtained from patients, the so-called “patients in a petri dish” as tools to investigate human genetic diseases for which there are no available effective treatment. Chapters embrace several examples of the use of iPS cell technology, a recent Nobel Prize-winning scientific breakthrough, to obtain patient-specific pluripotent cells from which many types of specialized cells involved in a particular disease can be generated, including psychiatric and neurodegenerative disorders, muscular dystrophies, laminopathies, among others. The text is a current and timely resource for postgraduate students, scientists and clinicians, interested in applications of this rapidly developing field of research in disease modeling, drug development, and emerging issues that it brings to regenerative medicine.

Author: Minoru Tomizawa
Publisher: BoD – Books on Demand
ISBN: 9535124714
Category : Science
Languages : en
Pages : 534

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Book Description
Pluripotent stem cells have distinct characteristics: self-renewal and the potential to differentiate into various somatic cells. In recent years, substantial advances have been made from basic science to clinical applications. The vast amount knowledge available makes obtaining concise yet sufficient information difficult, hence the purpose of this book. In this book, embryonic stem cells, induced pluripotent stem cells, and mesenchymal stem cells are discussed. The book is divided into five sections: pluripotency, culture methods, toxicology, disease models, and regenerative medicine. The topics covered range from new concepts to current technologies. Readers are expected to gain useful information from expert contributors.

Author: Raul Delgado-Morales
Publisher: Springer
ISBN: 331990695X
Category : Science
Languages : en
Pages : 426

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Book Description
This book looks at where stem cell technology is presently and how it is instrumental in advancing the field of disease modeling and cell transplantation. By focusing on major human disorders such as Alzheimer’s disease, cancer, and heart disorders, the book summarizes the major findings in the field of human stem cells and dissect the current limitations on our understanding of stem cells biology. The chapters focus on the genetics, genomics, epigenetics and physiology of stem cells models, together with technological advances on molecular biology such as CRISPR/Cas9 or epigenetic editing, that will be instrumental in the future of human disease modeling and treatment. In base of the limitations of current disease models and in front of the unmet necessity of finding therapeutical interventions for human disorders, the availability of stem cell technology has opened new doors for several fields. The unlimited self-renewal capacity and more extensive differentiation potential of stem cells offers a theoretically inexhaustible and replenishable source of any cell subtype. Since Professor Shinya Yamanaka described it, 10 years ago in his seminal paper, that somatic cells could be reprogrammed to inducible stem cells (iPSC) just by expressing four transcription factors, the field of has exploded, especially its applications in biomedical research.

Author: Phuc Van Pham
Publisher: Springer
ISBN: 3319980653
Category : Science
Languages : en
Pages : 190

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Book Description
This invaluable resource discusses insights ranging from basic biological mechanisms of various types of stem cells through the potential applications in the treatment of human diseases, including cancer and genetic disorders. These discoveries are placed within the structural context of tissue and developmental biology in sections dealing with recent advances in understanding different types of stem cell biology and their potential applications in tissue repair and regeneration and in the treatment different types of human cancer and genetic diseases or disorders. Stem Cells for Cancer and Genetic Disease Treatment and the other books in the Stem Cells in Clinical Applicationsseries will be invaluable to scientists, researchers, advanced students and clinicians working in stem cells, regenerative medicine or tissue engineering as well as cancer or genetics research.

Author: Institute of Medicine
Publisher: National Academies Press
ISBN: 0309170427
Category : Science
Languages : en
Pages : 112

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Book Description
Recent scientific breakthroughs, celebrity patient advocates, and conflicting religious beliefs have come together to bring the state of stem cell researchâ€"specifically embryonic stem cell researchâ€"into the political crosshairs. President Bush's watershed policy statement allows federal funding for embryonic stem cell research but only on a limited number of stem cell lines. Millions of Americans could be affected by the continuing political debate among policymakers and the public. Stem Cells and the Future of Regenerative Medicine provides a deeper exploration of the biological, ethical, and funding questions prompted by the therapeutic potential of undifferentiated human cells. In terms accessible to lay readers, the book summarizes what we know about adult and embryonic stem cells and discusses how to go about the transition from mouse studies to research that has therapeutic implications for people. Perhaps most important, Stem Cells and the Future of Regenerative Medicine also provides an overview of the moral and ethical problems that arise from the use of embryonic stem cells. This timely book compares the impact of public and private research funding and discusses approaches to appropriate research oversight. Based on the insights of leading scientists, ethicists, and other authorities, the book offers authoritative recommendations regarding the use of existing stem cell lines versus new lines in research, the important role of the federal government in this field of research, and other fundamental issues.

Author: Cell Press
Publisher: Elsevier
ISBN: 0127999167
Category : Science
Languages : en
Pages : 374

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Book Description
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: Safety and efficacy of stem cell treatments Stem-cell-based organ regeneration Integrating stem cells into personalized drug discovery Using pluripotent stem cells for treating neurological disease Engineering hematopoietic stem cells for innovative therapies 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. Provides timely overviews on a wide range of stem cell applications Offers insight from experts on the key opportunities and challenges facing the field Features reviews on genetic, cellular, and molecular aspects of stem-cell-based applications Includes articles originally published in Cell, Cell Stem Cell, Neuron, Trends in Molecular Medicine, and Trends in Biotechnology

Author: Arlene Chiu
Publisher: Springer Science & Business Media
ISBN: 1592594239
Category : Science
Languages : en
Pages : 455

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Book Description
A discussion of all the key issues in the use of human pluripotent stem cells for treating degenerative diseases or for replacing tissues lost from trauma. On the practical side, the topics range from the problems of deriving human embryonic stem cells and driving their differentiation along specific lineages, regulating their development into mature cells, and bringing stem cell therapy to clinical trials. Regulatory issues are addressed in discussions of the ethical debate surrounding the derivation of human embryonic stem cells and the current policies governing their use in the United States and abroad, including the rules and conditions regulating federal funding and questions of intellectual property.

Author: The Royal Society
Publisher: National Academies Press
ISBN: 0309671132
Category : Medical
Languages : en
Pages : 239

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Book Description
Heritable human genome editing - making changes to the genetic material of eggs, sperm, or any cells that lead to their development, including the cells of early embryos, and establishing a pregnancy - raises not only scientific and medical considerations but also a host of ethical, moral, and societal issues. Human embryos whose genomes have been edited should not be used to create a pregnancy until it is established that precise genomic changes can be made reliably and without introducing undesired changes - criteria that have not yet been met, says Heritable Human Genome Editing. From an international commission of the U.S. National Academy of Medicine, U.S. National Academy of Sciences, and the U.K.'s Royal Society, the report considers potential benefits, harms, and uncertainties associated with genome editing technologies and defines a translational pathway from rigorous preclinical research to initial clinical uses, should a country decide to permit such uses. The report specifies stringent preclinical and clinical requirements for establishing safety and efficacy, and for undertaking long-term monitoring of outcomes. Extensive national and international dialogue is needed before any country decides whether to permit clinical use of this technology, according to the report, which identifies essential elements of national and international scientific governance and oversight.

Author: Stephen H. Tsang
Publisher: Academic Press
ISBN: 0128178779
Category : Science
Languages : en
Pages : 138

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Book Description
CRISPR Genome Surgery in Stem Cells and Disease Tissues focuses uniquely on the clinical applications of CRISPR/Cas9 based technology. Topics include the latest advances in gene editing and its translational applications to various diseases, including retinal degenerative disease, recessively inherited diseases, and dominantly inherited diseases, to name a few. The book's target audience includes researchers, students, clinicians and the general public. This space that is not currently served by any existing resource, so this publication fills a gap in current literature. Provides a thorough review of CRISPR-Cas9, from discovery to therapy Covers the latest advances in gene editing and its translational applications to various diseases Written by global leaders in the fields of gene editing and stem cell therapy

Author: Deepak A. Lamba
Publisher: CRC Press
ISBN: 1315402173
Category : Medical
Languages : en
Pages : 280

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Book Description
One of the biggest challenges faced in medical research had been to create accurate and relevant models of human disease. A number of good animal models have been developed to understand the pathophysiology. However, not all of them reflect the human disorder, a classic case being Usher’s syndrome where the mutant mice do not have the same visual and auditory defects that patients face. There are others which have been even more difficult to model due to the multi-factorial nature of the condition and due to lack of discovery of a single causative gene such as age-related macular degeneration or Alzheimer’s syndrome. Thus a more relevant and accurate system will allow us to make better predictions on relevant therapeutic approaches. The discovery of human pluripotent stem cells in 1998 followed by the technological advances to reprogram somatic cells to pluripotent-stem cell-like cells in 2006 has completely revolutionized the way we can now think about modelling human development and disease. This now coupled with genome editing technologies such as TALENS and CRISPRs have now set us up to develop in vitro models both 2D as well as 3D organoids, which can more precisely reflect the disease in the patients. These combinatorial technologies are already providing us with better tools and therapeutics in drug discovery or gene therapy. This book summarizes both the technological advances in the field of generation of patient specific lines as well as various gene editing approaches followed by its applicability in various systems. The book will serve as a reference for the current state of the field as it: -Provides a comprehensive overview of the status of the field of patients derived induced pluripotent stem cells. -Describes the use of cardiac cells as a main featured component within the book. -Examines drug toxicity analysis as a working example throughout the book.