Cardiovascular Genome Editing

Genome Editing: A Practical Guide to Research and Clinical Applications is geared towards investigators interested in learning how to use CRISPR-Cas9-based technologies, with a focus on cardiovascular research and clinical applications. Covering a range of topics from the basics of genome editing to design considerations, to assessments and applications, this reference allows readers to get started and establish a full workflow from the beginning of the project to its full completion. With worked examples drawn from real-life experiments, as well as troubleshooting and pitfalls to avoid, the book serves as an essential reference for researchers and investigators in both cardiovascular and biomedical research. Help readers familiarise with the variety of genome-editing approaches that are being applied in cardiovascular research and medicine, i.e., both research applications and clinical applications Understand the use of genome editing through worked examples (based on real-life experiments) in which CRISPR-Cas9 is employed, online tools to design CRISPR-Cas9 reagents, methods to interpret data from genome-editing experiments, the downsides of genome-editing technology - both the scientific and ethical pitfalls to avoid Written in an easy-to-follow manner, guiding readers from the design of the project to its completion Includes unpublished and new methods

Translational Cardiometabolic Genomic Medicine, edited by Dr. Annabelle Rodriguez-Oquendo, is an important resource to postgraduate (medical, dental and graduate) students, postdoctoral fellows, basic scientists, and physician scientists seeking to understand and expand their knowledge base in the field of genomic medicine as it is applied to cardiometabolic diseases. This handbook integrates cutting-edge experimental approaches such as chromatin immunoprecipitation paired end tagging (CHIA-PET), to population studies such as the Multi-Ethnic Study of Atherosclerosis. It encompasses a range of book chapters that highlight bioinformatic approaches to better understanding functionality of the noncoding regions of the human genome to the use of molecular diagnostic testing in predicting increased risk of cardiovascular diseases. Where applicable, this reference also includes chapters related to therapeutic options specifically aligned to molecular targets. Provides comprehensive research on translational genomic medicine Explains state-of-the-art genome editing for stem cells and mouse models with significant relevance to human cardiometabolic disease Includes discussions on the functional effects of single nucleotide polymorphisms and cardiometabolic diseases, stratified by sex and race Encompasses a range of book chapters that highlight bioinformatic approaches to better understanding functionality of the noncoding regions of the human genome

Cardiovascular Diseases: Genetic Susceptibility, Environmental Factors and Their Interaction covers the special heritability characteristics and identifying genetic and environmental contributions to cardiovascular health. This important reference provides an overview of the genetic basis of cardiovascular disease and its risk factors. Included are important topics, ranging from lifestyle choices, risk factors, and exposure, to pollutants and chemicals. Also covered are the influences of Mendelian traits and familial aggregation and the interactions and interrelationships between genetics and environmental factors which, when compared, provide a sound understanding of the interplay between inherited and acquired risk factors. The book provides a much needed reference for this rapidly growing field of study. By combining the latest research within the structured chapters of this reference, a better understanding of genetic and environmental contribution to cardiovascular disease is found, helping to substantiate further investigations in the field and design prevention and treatment strategies. Provides an overview of the genetic basis of cardiovascular disease and its risk factors Reviews several large population-based studies which indicate that exposure to several environmental factors may increase CVD morbidity and mortality, exploring the plausibility of this association by data from animal studies Reflects on future studies to help understanding the role of genes and environmental factors in the development and progression of cardiovascular disease

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.

Until recently, a modest knowledge of genetics was more than adequate for the daily practice of clinical cardiology, but advances in genetics and genomics are changing cardiovascular medicine in fundamental ways. The identification of the genetic basis of several forms of dyslipidemia, hypertension, diabetes, cardiomyopathies, and vascular diseases signalled the new importance of genetics in clinical medicine. In this timely volume, Drs. Dzau and Liew – both pioneers in the area – help cardiologists understand: • how cardiovascular genetics may remodel the way cardiovascular medicine is practiced • what material has immediate relevance to the practicing clinician • how to incorporate genetics and genomics in your practice to ensure up-to-date patient care The book opens with introductory chapters, then discusses: • cardiovascular single gene disorders • cardiovascular polygenic disorders • therapies and applications Outstanding contributors write on their areas of expertise, making Cardiovascular Genetics and Genomics for the Cardiologist both authoritative and comprehensive. If you want to gain a better appreciation of how genetics and genomics are already shaping current practice and may potentially revolutionize cardiology, look no further than this dependable reference.

A complete guide to endonuclease-based genomic engineering, from basic science to application in disease biology and clinical treatment.

This detailed book provides methodological information on cardiac gene delivery, from classic to state-of-the-art technologies and techniques. Efficient, cardiac-specific, and safe vectors, as well as refined vector delivery methods, are key for successful cardiac gene transfer and eventually for improving patients’ outcomes. Newer vectors and more efficient vector delivery methods have the potential to dramatically improve gene transduction efficacy, while novel gene manipulation techniques enforce the therapeutic power and broaden disease targets. Written for the highly successful Methods in Molecular Biology series, chapters include introductions to their respective topics, lists of the necessary materials and reagents, step-by-step, readily reproducible laboratory protocols, and tips on troubleshooting and avoiding known pitfalls. Authoritative and practical, Cardiac Gene Therapy: Methods and Protocols serves as a valuable tool for molecular biologists and physiologists in the cardiology field conducting cardiac gene transfer research, which will ultimately lead to further advancements in the vital field.

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

This volume of the esteemed Progress in Molecular Biology and Translational Science series focuses on cutting-edge research related to the genetics of cardiovascular disease. Written by top experts in the field, this volume covers a range of topics, including: Genetic models of atherosclerosis MicroRNAs in cardiovascular disease The role of the transcription factor KLF2 in vascular development and disease Zebrafish as models of cardiovascular disease and hematopoietic development Progress in Molecular Biology and Translational Science provides a forum for discussion of new discoveries, approaches, and ideas in molecular biology. It contains contributions from leaders in their fields and abundant references. The authors are all experts in the field The reviews are in themselves comprehensive Each topic has the potential to lead to advances in therapy of cardiovascular disease

This timely volume explores the use of CRISPR-Cas9 for genome editing, presenting cutting-edge techniques and their applications in treatment of disease. The chapters describe latest methods such as use of targetable nucleases, investigation of the non-coding genome, mouse genome editing, increasing of knock-in efficiency in mouse zygotes, and generation of reporter stem cells; the text contextualizes these methods in treatment of cardiovascular disease, diabetes mellitus, retinitis pigmentosa, and others. The final chapters round out the book with a discussion of controversies and future directions. Genome Editing is an essential, of-the-moment contribution to this rapidly growing field. Drawing from a wealth of international perspectives, it presents novel techniques and applications for the engineering of the human genome. This book is essential reading for all clinicians and researchers in stem cells, regenerative medicine, genomics, biochemical and biomedical engineering- especially those interested in learning more about genome editing and applying it in a targeted, specific way.

The properties of human pluripotent stem cells (hPSCs) make them uniquely suited to address this challenge - they retain a normal human genome in culture, can be genetically modified, and can be differentiated into multiple cell types. The work presented in this thesis demonstrates the use of hPSCs and genome editing technology to generate human disease models in vitro. We developed a genome editing system optimized for hPSCs that can be used to efficiently generate hPSC lines with targeted genetic modifications. Modified and isogenic control hPSCs are then differentiated into a relevant cell type for phenotypic characterization. We applied this approach to investigate the functional effects of human genetic variation underlying plasma lipid concentrations.

Precision Medicine for Investigators, Practitioners and Providers addresses the needs of investigators by covering the topic as an umbrella concept, from new drug trials to wearable diagnostic devices, and from pediatrics to psychiatry in a manner that is up-to-date and authoritative. Sections include broad coverage of concerning disease groups and ancillary information about techniques, resources and consequences. Moreover, each chapter follows a structured blueprint, so that multiple, essential items are not overlooked. Instead of simply concentrating on a limited number of extensive and pedantic coverages, scholarly diagrams are also included. Provides a three-pronged approach to precision medicine that is focused on investigators, practitioners and healthcare providers Covers disease groups and ancillary information about techniques, resources and consequences Follows a structured blueprint, ensuring essential chapters items are not overlooked

"A subject collection from Cold Spring Harbor perspectives in medicine."

The development of the cardiovascular system is a rapidly advancing area in biomedical research, now coupled with the burgeoning field of cardiac regenerative medicine. A lucid understanding of these fields is paramount to reducing human cardiovascular diseases of both fetal and adult origin. Significant progress can now be made through a comprehensive investigation of embryonic development and its genetic control circuitry. Heart Development and Regeneration, written by experts in the field, provides essential information on topics ranging from the evolution and lineage origins of the developing cardiovascular system to cardiac regenerative medicine. A reference for clinicians, medical researchers, students, and teachers, this publication offers broad coverage of the most recent advances. Volume One discusses heart evolution, contributing cell lineages; model systems; cardiac growth; morphology and asymmetry; heart patterning; epicardial, vascular, and lymphatic development; and congenital heart diseases. Volume Two includes chapters on transcription factors and transcriptional control circuits in cardiac development and disease; epigenetic modifiers including microRNAs, genome-wide mutagenesis, imaging, and proteomics approaches; and the theory and practice of stem cells and cardiac regeneration. Authored by world experts in heart development and disease New research on epigenetic modifiers in cardiac development Comprehensive coverage of stem cells and prospects for cardiac regeneration Up-to-date research on transcriptional and proteomic circuits in cardiac disease Full-color, detailed illustrations

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.

Genomic and Precision Medicine: Oncology, Third Edition focuses on the applications of genome discovery as research points to personalized cancer therapies. Each chapter is organized to cover the application of genomics and personalized medicine tools and technologies to a) Risk Assessment and Susceptibility, b) Diagnosis and Prognosis, c) Pharmacogenomics and Precision Therapeutics, and d) Emerging and Future Opportunities in the field. Provides a comprehensive volume written and edited by oncology genomic specialists for oncology health providers Includes succinct commentary and key learning points that will assist providers with their local needs for implementation of genomic and personalized medicine into practice Presents an up-to-date overview on major opportunities for genomic and personalized medicine in practice Covers case studies that highlight the practical use of genomics in the management of patients

Nucleic Acid Nanotheranostics: Biomedical Applications offers a comprehensive overview of improvements and new trends in fabrication of nanostructures as theranostic multifunctional carriers in gene therapy. With a strong focus on medical applications (comprising diagnosis, therapy and imaging), the book also examines gene therapy in an individual patient’s cells or tissues to treat genetic diseases. Sections cover Biomedical and Diagnostic applications of Nucleic Acids, Biologic and Synthetic Advanced Nanostructures for nucleic acid delivery, and important considerations of nanomedicine. This book is a valuable guide for materials scientists, physicians, chemists and engineers, but is also ideal for clinicians wishing to expand their knowledge. Provides a unique source of knowledge (theoretical as well as practical) on nanotheranostic materials for gene therapy at all levels and related scientific areas Covers the pros and cons related to viral and nanomaterial-based delivery of nucleic acids in terms of biosafety, carrier selection, synthesis and bioimaging Presents the only book to include an analysis of nanoformulations approved for clinical use

Genome Engineering via CRISPR-Cas9 Systems presents a compilation of chapters from eminent scientists from across the globe who have established expertise in working with CRISPR-Cas9 systems. Currently, targeted genome engineering is a key technology for basic science, biomedical and industrial applications due to the relative simplicity to which they can be designed, used and applied. However, it is not easy to find relevant information gathered in a single source. The book contains a wide range of applications of CRISPR in research of bacteria, virus, algae, plant and mammalian and also discusses the modeling of drosophila, zebra fish and protozoan, among others. Other topics covered include diagnosis, sensor and therapeutic applications, as well as ethical and regulatory issues. This book is a valuable source not only for beginners in genome engineering, but also researchers, clinicians, stakeholders, policy makers, and practitioners interested in the potential of CRISPR-Cas9 in several fields. Provides basic understanding and a clear picture on how to design, use and implement the CRISPR-Cas9 system in different organisms Explains how to create an animal model for disease research and screening purposes using CRISPR Discusses the application of CRISPR-Cas9 systems in basic sciences, biomedicine, virology, bacteriology, molecular biology, neurology, cancer, industry, and many more