Reprogramming the Genome  CRISPR Cas based Human Disease Therapy Book

Reprogramming the Genome CRISPR Cas based Human Disease Therapy


  • Author : Anonim
  • Publisher : Academic Press
  • Release Date : 2021-06-12
  • Genre: Science
  • Pages : 390
  • ISBN 10 : 9780323853248

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Reprogramming the Genome CRISPR Cas based Human Disease Therapy Excerpt :

Reprogramming the Genome: CRISPR-Cas-based Human Disease Therapy, presents the collation of chapters written by eminent scientists worldwide. CRISPR-Cas9 is a key technology for targeted genome editing and regulation in a number of organisms including mammalian cells. It is a rapid, simple, and cost-effective solution. CRISPR-Cas system has recently gained much scientific and public attention. This volume covers CRISPR-Cas9 based mammalian genome editing, creating disease models, cancer therapy, neurological, heredity, blood disorders, defective gene correction, stem cells therapy, epigenetic modifications, patents, ethics, biosafety and regulatory issues challenges and opportunities. This book is a key source of information on mammalian genome editing available in a single volume. This book will be useful for beginners in mammalian genome editing and also students, researchers, scientists, policymakers, clinicians and stakeholders interested in genome editing in several areas. Offers basic understanding and a clear picture of mammalian genome editing through CRISPR-Cas systems Discusses how to create mammalian disease models, stem cell modification, epigenetic modifications, correction of defective gene in blood disorders, heredity, neurological disorders and many more Discusses the application of CRISPR-Cas9 systems in basic sciences, biomedicine, molecular biology, translational sciences, neurobiology, neurology, cancer, stem cells, and many more

Curing Genetic Diseases through Genome Reprogramming Book

Curing Genetic Diseases through Genome Reprogramming


  • Author : Anonim
  • Publisher : Academic Press
  • Release Date : 2021-06-24
  • Genre: Science
  • Pages : 558
  • ISBN 10 : 9780323853026

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Curing Genetic Diseases through Genome Reprogramming Excerpt :

Curing Genetic Diseases through Genome Reprogramming, Volume 182 captures an historic moment in the field of gene therapy—the dawn of a new age in which the dream of curing genetic diseases has become realizable. The volume presents the most clinically advanced gene therapy and genome editing approaches for the treatment of genetic diseases in specific organs, including difficult therapeutic targets, futuristic ideas of genetic interventions, and large scale human genome repair. An initial chapter addresses the complex ethical aspects involved in the very idea of modifying the human genome. Provides a comprehensive view of gene therapy and genome editing technologies, including epigenetic editing Describes the state-of-the-art and future directions for the treatment of genetic diseases, also considering economical aspects Presents chapters that each give a thorough review of a specific disease, target organ or visionary approach, including ethical considerations

CRISPR Cas Systems Book

CRISPR Cas Systems


  • Author : Rodolphe Barrangou
  • Publisher : Springer Science & Business Media
  • Release Date : 2012-12-13
  • Genre: Science
  • Pages : 302
  • ISBN 10 : 9783642346576

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CRISPR Cas Systems Excerpt :

CRISPR/Cas is a recently described defense system that protects bacteria and archaea against invasion by mobile genetic elements such as viruses and plasmids. A wide spectrum of distinct CRISPR/Cas systems has been identified in at least half of the available prokaryotic genomes. On-going structural and functional analyses have resulted in a far greater insight into the functions and possible applications of these systems, although many secrets remain to be discovered. In this book, experts summarize the state of the art in this exciting field.

Reprogramming the Genome  Applications of CRISPR Cas in non mammalian systems part A Book

Reprogramming the Genome Applications of CRISPR Cas in non mammalian systems part A


  • Author : Anonim
  • Publisher : Academic Press
  • Release Date : 2021-03-28
  • Genre: Science
  • Pages : 242
  • ISBN 10 : 9780323853224

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Reprogramming the Genome Applications of CRISPR Cas in non mammalian systems part A Excerpt :

Reprogramming the Genome: Applications of CRISPR-Cas in Non-mammalian Systems, Part A presents a collation of chapters written by global, eminent scientists. CRISPR-Cas9 system is an RNA-mediated immune system of bacteria and archaea that protects from bacteriophage infections. It is one of the revolutionized technologies to uplift biology to the next stages. Chapters in this release include An Introduction and applications of CRISPR-Cas Systems, History, evolution and classification of CRISPR-Cas associated systems, CRISPR based bacterial genome editing and removal of pathogens, CRISPR based genome editing and removal of human viruses, CRISPR based development of RNA editing and diagnostic platform, and much more. Additional sections cover Genome engineering in insects for control of vector borne diseases, Development of insect cell line using CRISPR technology, CRISPRing protozoan parasites to better understand the biology of diseases, CRISPR based genome editing of Caenorhabditis elegans, and a variety of other important topics. Offers a basic understanding and clear picture of genome editing CRISPR-Cas systems in different organisms Explains how to create an animal model for disease diagnosis/research and reprogram CRISPR for removal of virus, bacteria, fungi, protozoan, and many more Discusses the advances, patents, applications, challenges and opportunities in CRISPR-Cas9 systems in basic sciences, biomedicine, virology, bacteriology, molecular biology, and many more

CRISPR Genome Surgery in Stem Cells and Disease Tissues Book

CRISPR Genome Surgery in Stem Cells and Disease Tissues


  • Author : Stephen H. Tsang
  • Publisher : Academic Press
  • Release Date : 2021-10-20
  • Genre: Science
  • Pages : 136
  • ISBN 10 : 9780128178775

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CRISPR Genome Surgery in Stem Cells and Disease Tissues Excerpt :

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

Reprogramming the Genome  Applications of CRISPR Cas in non mammalian systems part B Book

Reprogramming the Genome Applications of CRISPR Cas in non mammalian systems part B


  • Author : Anonim
  • Publisher : Academic Press
  • Release Date : 2021-04-29
  • Genre: Science
  • Pages : 198
  • ISBN 10 : 9780323852982

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Reprogramming the Genome Applications of CRISPR Cas in non mammalian systems part B Excerpt :

Reprogramming the Genome: Applications of CRISPR-Cas in Non-mammalian Systems Part B, represents the collation of chapters written by eminent scientists worldwide. CRISPR-Cas9 system is an RNA-mediated immune system of bacteria and archaea that protects from bacteriophage infections. It is one of the revolutionized technologies to uplift biology to the next stages. It is a simple, rapid, precise, and cost-effective tool for genome editing and regulation of a wide range of organisms. It has gained scientific and public attention worldwide. This volume mainly covers insect cell line, protozoans, zebrafish, drosophila, CRISPRi, patents as well as technology transfer, and many more. This book is a key source of information available in a single volume. This book will be useful for not only beginners in genome engineering, but also students, researchers, scientists, policymakers, and stakeholders interested in harnessing the potential of reprogramming of the genomes in several areas. Offers basic understanding and a clear picture of genome editing CRISPR-Cas systems in different organisms Explains how to create an animal model for disease diagnosis/research and reprogram CRISPR for insect cell line, protozoans, zebrafish, drosophila, and many more Discusses the advances, patents, applications, challenges and opportunities in CRISPR-Cas9 systems in basic sciences, biomedicine, molecular biology and many more

Heritable Human Genome Editing Book

Heritable Human Genome Editing


  • Author : The Royal Society
  • Publisher : National Academies Press
  • Release Date : 2021-01-16
  • Genre: Medical
  • Pages : 238
  • ISBN 10 : 9780309671132

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Heritable Human Genome Editing Excerpt :

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.

CRISPR Cas Enzymes Book

CRISPR Cas Enzymes


  • Author : Anonim
  • Publisher : Academic Press
  • Release Date : 2019-01-25
  • Genre: Science
  • Pages : 451
  • ISBN 10 : 9780128167618

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CRISPR Cas Enzymes Excerpt :

CRISPR-Cas Enzymes, Volume 616, the latest release in the Methods in Enzymology series, continues the legacy of this premier serial with quality chapters authored by leaders in the field. Topics covered in this release include CRISPR bioinformatics, A method for one-step assembly of Class 2 CRISPR arrays, Biochemical reconstitution and structural analysis of ribonucleoprotein complexes in Type I-E CRISPR-Cas systems, Mechanistic dissection of the CRISPR interference pathway in Type I-E CRISPR-Cas system, Site-specific fluorescent labeling of individual proteins within CRISPR complexes, Fluorescence-based methods for measuring target interference by CRISPR-Cas systems, Native State Structural Characterization of CRISRP Associated Complexes using Mass Spectrometry, and more. Provides the authority and expertise of leading contributors from an international board of authors Presents the latest release in the Methods in Enzymology series Updated release includes the latest information on the CRISPR-Cas Enzymes

Human Genome Editing Book

Human Genome Editing


  • Author : National Academies of Sciences, Engineering, and Medicine
  • Publisher : National Academies Press
  • Release Date : 2017-08-13
  • Genre: Medical
  • Pages : 328
  • ISBN 10 : 9780309452885

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Human Genome Editing Excerpt :

Genome editing is a powerful new tool for making precise alterations to an organism's genetic material. Recent scientific advances have made genome editing more efficient, precise, and flexible than ever before. These advances have spurred an explosion of interest from around the globe in the possible ways in which genome editing can improve human health. The speed at which these technologies are being developed and applied has led many policymakers and stakeholders to express concern about whether appropriate systems are in place to govern these technologies and how and when the public should be engaged in these decisions. Human Genome Editing considers important questions about the human application of genome editing including: balancing potential benefits with unintended risks, governing the use of genome editing, incorporating societal values into clinical applications and policy decisions, and respecting the inevitable differences across nations and cultures that will shape how and whether to use these new technologies. This report proposes criteria for heritable germline editing, provides conclusions on the crucial need for public education and engagement, and presents 7 general principles for the governance of human genome editing.

iPS Cells for Modelling and Treatment of Human Diseases Book

iPS Cells for Modelling and Treatment of Human Diseases


  • Author : Michael J. Edel
  • Publisher : MDPI
  • Release Date : 2018-10-02
  • Genre: Medical
  • Pages : 422
  • ISBN 10 : 9783038421221

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iPS Cells for Modelling and Treatment of Human Diseases Excerpt :

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

CRISPR in Animals and Animal Models Book

CRISPR in Animals and Animal Models


  • Author : Anonim
  • Publisher : Academic Press
  • Release Date : 2017-11-10
  • Genre: Science
  • Pages : 148
  • ISBN 10 : 9780128125076

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CRISPR in Animals and Animal Models Excerpt :

CRISPR in Animals and Animal Models, Volume 152, the latest release in the Progress in Molecular Biology and Translational Science series, explores the genome editing CRISPR system in cells and animal models, its applications, the uses of the CRISPR system, and the past, present and future of CRISPR genome editing. Topics of interest in this updated volume include a section on CRISPR history, The genome editing revolution, Programming CRISPR and its applications, CRISPR Delivery methods, CRISPR libraries and screening, CRISPR investigation in haploid cells, CRISPR in the generation of transgenic animals, CRISPR therapeutics, and Promising strategies and present challenges. Accessible to students and researchers alike Written by leading authorities in the field

Engineering Efficient and Safe in Situ Genome Regulation Via CRISPR Cas9 for Enabling Gene Therapies Book

Engineering Efficient and Safe in Situ Genome Regulation Via CRISPR Cas9 for Enabling Gene Therapies


  • Author : Ana Maria Moreno
  • Publisher : Unknown
  • Release Date : 2019
  • Genre: Uncategoriezed
  • Pages : 175
  • ISBN 10 : OCLC:1119669279

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Engineering Efficient and Safe in Situ Genome Regulation Via CRISPR Cas9 for Enabling Gene Therapies Excerpt :

While the genetic and pathogenic basis of human diseases continues to grow, translation is currently bottlenecked by lack of tools and technologies to administer and evaluate corresponding gene-based therapeutics. Consequently, development of safe and efficient in vivo gene transfer platforms, coupled with emerging genome and epigenome engineering tools, will transform our ability to target a range of human diseases. In this regard, the holy grail of in vivo genome engineering is the ability to achieve the trifecta of: 1) efficient and safe delivery; 2) temporally regulatable and tunable payload delivery; and 3) immune stealth to minimize dosage & enable re-administration of nucleic acid or protein therapeutics. Towards this, the objective of this dissertation was to develop a platform to enable efficacious in vivo genome and epigenome engineering with a focus on enabling in situ therapeutic efficacy. The studies in this dissertation are independent bodies of work that explore the optimization and engineering of CRISPR-Cas9 systems to bring these one step closer to their eventual translation into the clinic. Towards these, I first developed a robust and generalizable platform for in situ genome editing and regulation via AAV CRISPR-Cas9. Towards this, I utilized split-Cas9 systems to develop a modular adeno-associated viral (AAV) vector platform for CRISPR-Cas9 delivery to enable the full spectrum of targeted in situ gene regulation functionalities, demonstrating robust transcriptional repression (up to 80%) and activation (up to 6-fold) of target genes in cell culture and mice. We also applied our platform for targeted in vivo gene-repression-mediated gene therapy for retinitis pigmentosa. Specifically, we engineered targeted repression of Nrl, a master regulator of rod photoreceptor determination, and demonstrated Nrl knockdown mediates in situ reprogramming of rod cells into cone-like cells that are resistant to retinitis pigmentosa-specific mutations, with conco

Molecular and Cellular Therapies for Motor Neuron Diseases Book

Molecular and Cellular Therapies for Motor Neuron Diseases


  • Author : Nicholas M Boulis
  • Publisher : Academic Press
  • Release Date : 2017-01-18
  • Genre: Psychology
  • Pages : 336
  • ISBN 10 : 9780128025246

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Molecular and Cellular Therapies for Motor Neuron Diseases Excerpt :

Molecular and Cellular Therapies for Motor Neuron Diseases discusses the basics of the diseases, also covering advances in research and clinical trials. The book provides a resource for students that will help them learn the basics in a detailed manner that is required for scientists and clinicians. Users will find a comprehensive overview of the background of Amyotrophic Lateral Sclerosis (ALS/Lou Gehrig’s Disease) and Spinal Muscular Atrophy (SMA), along with the current understanding of their genetics and mechanisms. In addition, the book details gene and cell therapies that have been developed and their translation to clinical trials. Provides an overview of gene and cell therapies for amyotrophic lateral sclerosis (ALS) and other motor neuron diseases Edited by a leading Neurosurgeon and two research scientists to promote synthesis between basic neuroscience and clinical relevance Presents a great resource for researchers and practitioners in neuroscience, neurology, and gene and cell therapy

Nutritional Epigenomics Book

Nutritional Epigenomics


  • Author : Anonim
  • Publisher : Academic Press
  • Release Date : 2019-07-20
  • Genre: Medical
  • Pages : 478
  • ISBN 10 : 9780128173107

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Nutritional Epigenomics Excerpt :

Nutritional Epigenomics offers a comprehensive overview of nutritional epigenomics as a mode of study, along with nutrition’s role in the epigenomic regulation of disease, health and developmental processes. Here, an expert team of international contributors introduces readers to nutritional epigenomic regulators of gene expression, our diet’s role in epigenomic regulation of disease and disease inheritance, caloric restriction and exercise as they relate to recent epigenomic findings, and the influence of nutritional epigenomics over circadian rhythms, aging and longevity, and fetal health and development, among other processes. Disease specific chapters address metabolic disease (obesity and diabetes), cancer, and neurodegeneration, among other disorders. Diet-gut microbiome interactions in the epigenomic regulation of disease are also discussed, as is the role of micronutrients and milk miRNAs in epigenetic regulation. Finally, chapter authors examine ongoing discussions of race and ethnicity in the social-epigenomic regulation of health and disease. Empowers the reader to employ nutritional epigenomics approaches in their own research Discusses the latest topics in nutritional epigenomics in the regulation of aging, circadian rhythm, inheritance and fetal development, as well as metabolism and disease Offers a full grounding in epigenetic reprogramming and nutritional intervention in the treatment and prevention of disease, as informed by population-based studies