Modeling Human Genetic Diseases Using Human Embryonic Stem Cells

Modeling Human Genetic Diseases Using Human Embryonic Stem Cells PDF Author: Achia Urbach
Publisher:
ISBN:
Category : Embryonic stem cells
Languages : en
Pages : 204

Get Book Here

Book Description

Stem Cells in Modeling Human Genetic Diseases

Stem Cells in Modeling Human Genetic Diseases PDF Author: Mayana Zatz
Publisher: Springer
ISBN: 3319183141
Category : Science
Languages : en
Pages : 157

Get Book Here

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.

Modeling Human Genetic Diseases Using Human Embryonic Stem Cells

Modeling Human Genetic Diseases Using Human Embryonic Stem Cells PDF Author: Achia Urbach
Publisher:
ISBN:
Category : Embryonic stem cells
Languages : en
Pages : 204

Get Book Here

Book Description


Pluripotent Stem Cells

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

Get Book Here

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.

Heritable Human Genome Editing

Heritable Human Genome Editing PDF Author: The Royal Society
Publisher: National Academies Press
ISBN: 0309671132
Category : Medical
Languages : en
Pages : 239

Get Book Here

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.

Stem Cells and the Future of Regenerative Medicine

Stem Cells and the Future of Regenerative Medicine PDF Author: Institute of Medicine
Publisher: National Academies Press
ISBN: 0309170427
Category : Science
Languages : en
Pages : 112

Get Book Here

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.

Examining the State of the Science of Mammalian Embryo Model Systems

Examining the State of the Science of Mammalian Embryo Model Systems PDF Author: National Academies of Sciences, Engineering, and Medicine
Publisher: National Academies Press
ISBN: 0309676681
Category : Medical
Languages : en
Pages : 155

Get Book Here

Book Description
Because of the recent advances in embryo modeling techniques, and at the request of the Office of Science Policy in the Office of the Director at the National Institutes of Health, the National Academies of Sciences, Engineering, hosted a 1-day public workshop that would explore the state of the science of mammalian embryo model systems. The workshop, which took place on January 17, 2020, featured a combination of presentations, panels, and general discussions, during which panelists and participants offered a broad range of perspectives. Participants considered whether embryo model systems - especially those that use nonhuman primate cells - can be used to predict the function of systems made with human cells. Presentations provided an overview of the current state of the science of in vitro development of human trophoblast. This publication summarizes the presentation and discussion of the workshop.

Stem Cells for Modeling Human Disease

Stem Cells for Modeling Human Disease PDF Author: Martín F
Publisher:
ISBN:
Category : Science
Languages : en
Pages :

Get Book Here

Book Description
Human pluripotent stem cells (PSCs) in the form of human embryonic stem cells (hESCs) or induced pluripotent stem cells (iPSCs) are capable of growing indefinitely in vitro, maintaining their capacity to differentiate into the three primary germ layers: mesoderm, endoderm and ectoderm. Different protocols have been developed to differentiate PSCs into almost any cellular type with different degree of success. This technology has allowed scientists to use patient-derived iPSCs to study the physiopathology of the disease by analyzing the phenotype of the cells derived from these iPSCs. However, control iPSCs obtained from healthy individuals will always have different genomic environment than patient's iPSCs, making it difficult the interpretation of the cells phenotype. The recent appearance of specific nucleases [zinc-finger nucleases (ZFNs), the transcription activator-like effector nucleases (TALENs) and the clustered regularly interspaced short palindromic repeats (CRISPR)] has made it possible to edit the genome of PSCs. We can now generate syngeneic hESCs or iPSCs harboring the desired mutation and comparing the emerging cells with those derived from genetically identical PSCs that will differ only in the mutated gene. In this chapter, we summarize the progress made in this field and discuss the different approaches that have been used recently for the generation of syngeneic human pluripotent cellular models for different pathologies.

iPS Cells for Modelling and Treatment of Human Diseases

iPS Cells for Modelling and Treatment of Human Diseases PDF Author: Michael J. Edel
Publisher: MDPI
ISBN: 3038421227
Category : Electronic book
Languages : en
Pages : 422

Get Book Here

Book Description
This book is a printed edition of the Special Issue "iPS Cells for Modelling and Treatment of Human Diseases" that was published in JCM

Human iPSC-derived Disease Models for Drug Discovery

Human iPSC-derived Disease Models for Drug Discovery PDF Author: Markus H. Kuehn
Publisher: Springer Nature
ISBN: 3031423496
Category : Medical
Languages : en
Pages : 331

Get Book Here

Book Description
Since their development a decade ago, human induced pluripotent stem cells (iPSC) have revolutionized the study of human disease, given rise to regenerative medicine technologies, and provided exceptional opportunities for pharmacologic research. These cells provide an essentially unlimited supply of cell types that are difficult to obtain from patients, such as neurons or cardiomyocytes, or are difficult to maintain in primary cell culture. iPSC can be obtained from patients afflicted with a particular disease but, in combination with recently developed gene editing techniques, can also be modified to generate disease models. Moreover, the new techniques of 3 Dimensional printing and materials science facilitate the generation of organoids that can mirror organs under disease conditions. These properties make iPSC powerful tools to study how diseases develop and how they may be treated. In addition, iPSC can also be used to treat conditions in which the target cell population has been lost and such regenerative approaches hold great promise for currently untreatable diseases, including cardiac failure or photoreceptor degenerations.

Stem Cell Genetics for Biomedical Research

Stem Cell Genetics for Biomedical Research PDF Author: Raul Delgado-Morales
Publisher: Springer
ISBN: 331990695X
Category : Science
Languages : en
Pages : 426

Get Book Here

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.