Download or read book Reprogramming the Genome CRISPR Cas based Human Disease Therapy written by and published by Academic Press. This book was released on 2021-06-12 with total page 394 pages. Available in PDF, EPUB and Kindle. Book 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

Download or read book CRISPR Cas9 Based Genome Editing for Treating Genetic Disorders and Diseases written by Luis María Vaschetto and published by CRC Press. This book was released on 2022-01-31 with total page 268 pages. Available in PDF, EPUB and Kindle. Book excerpt: The CRISPR-Cas9 genome-editing system is creating a revolution in the science world. In the laboratory, CRISPR-Cas9 can efficiently be used to target specific genes, correct mutations and regulate gene expression of a wide array of cells and organisms, including human cells. CRISPR-/Cas9 Based Genome Editing for Treating Genetic Disorders and Diseases is a unique reading material for college students, academicians, and other health professionals interested in learning about the broad range of applications of CRISPR/Cas9 genetic scissors. Some topics included in this book are: the role of the CRISPR/Cas9 system in neuroscience, gene therapy, epigenome editing, genome mapping, cancer, virus infection control strategies, regulatory challenges and bioethical considerations.

Download or read book Curing Genetic Diseases through Genome Reprogramming written by and published by Academic Press. This book was released on 2021-06-24 with total page 560 pages. Available in PDF, EPUB and Kindle. Book 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

Download or read book CRISPR Cas Systems written by Rodolphe Barrangou and published by Springer Science & Business Media. This book was released on 2012-12-13 with total page 300 pages. Available in PDF, EPUB and Kindle. Book 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.

Download or read book CRISPR Cas Based Genome Editing for Treating Human Diseases Part A written by and published by Elsevier. This book was released on 2024-09-11 with total page 310 pages. Available in PDF, EPUB and Kindle. Book excerpt: CRISPR-Cas-Based Genome Editing for Treating Human Diseases-Part A, Volume 208 represents CRISPR-Cas systems for genome editing. Currently, CRISPR-Cas systems are proven a key technology for targeted genome editing, which is acting as a simple, rapid, and cost-effective solution. CRISPR-Cas9 system is being used in microbial genome editing, mammalian genome editing, disease models, and more. It has shown potential in human disease treatment. However, it is not easy to find CRISPR-Cas systems genome editing in a single source.This volume offers CRISPR-Cas systems for human diseases, bacterial disease, gut microbiome editing, viral disease, protozoan disease, fungal disease, stem cell therapy, CRISPR in epigenetics, CRISPR in cancer, autoimmune and blood disorders. In addition, it highlights a number of aspects of the CRISPR-Cas systems that can help the basic understanding of students, researchers, clinicians, entrepreneurs, and stakeholders to perform their research with great interest. - Updares on CRISPR-Cas systems for human diseases, bacterial disease, gut microbiome editing, viral disease, protozoan disease and fungal disease - Discusses stem cell therapy, CRISPR in epigenetics, CRISPR in cancer, autoimmune and blood disorders - Includes commentary on CRISPR in autoimmune and blood disorders

Download or read book Reprogramming the Genome Applications of CRISPR Cas in non mammalian systems part A written by and published by Academic Press. This book was released on 2021-03-28 with total page 244 pages. Available in PDF, EPUB and Kindle. Book 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

Download or read book Heritable Human Genome Editing written by The Royal Society and published by National Academies Press. This book was released on 2021-01-16 with total page 239 pages. Available in PDF, EPUB and Kindle. Book 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.

Download or read book Anticipatory Ethics and The Use of CRISPR in Humans written by Michael W. Nestor and published by Springer Nature. This book was released on 2022-05-20 with total page 156 pages. Available in PDF, EPUB and Kindle. Book excerpt: The future of gene editing in humans will involve the use of CRISPR. How we think about the combination of the scientific, ethical, and moral aspects of this technology is paramount to the success or failure of CRISPR in humans. Unfortunately, the current scientific discussion around CRISPR in humans has left ethics trailing behind due to the rapid pace of innovation. New modes of ethics and stakeholder participation are needed to keep pace with rapid scientific advances and provide the necessary policy and ethical frameworks necessary to help CRISPR flourish as an important health care tool to treat human disease. This requires intense interdisciplinary collaboration and discussion between scientists and philosophers, policymakers and legal scholars, and the public. Dr. Michael W. Nestor (a neuroscientist who actively uses CRISPR in pre-clinical research) and Professor Richard Wilson (a philosopher who focuses on anticipatory ethics) set out to develop a new ethical approach considering the use of CRISPR in human targeted therapies. The field of anticipatory ethics is uniquely poised to tackle questions in fast-evolving technical areas where the pace of innovation outstrips traditional philosophical approaches. Furthermore, because of its “anticipatory” nature, this type of analysis provides the opportunity to look ahead and into the future concerning potential uses of CRISPR in humans, uses that are not currently possible. Nestor and Wilson collaborate both scientifically and philosophically in this book to forecast potential outcomes as the scientific and medical community goes beyond using CRISPR to correct genes that underlie diseases where a single gene is involved. Instead, Nestor and Wilson envision CRISPR in complex, multigenic disorders with a specific focus on the use of CRISPR to edit genes involved in mental traits like IQ or other cognitive characteristics. They argue that the use of CRISPR to modify genes that are potentially important for mental traits represents a particular category for special consideration from scientists, policymakers, the public, and other stakeholders. Nestor and Wilson explain why using CRISPR to alter mental states is very different from treating a disease like cancer by combining the latest scientific advancements with anticipatory ethics and philosophical phenomenology. Their analysis considers the role that mental states play in personhood and the lived experience-as genes that can change mental/cognitive attributes like IQ have wide-ranging effects on the lived experience in ways that are categorically different from other attributes. This book was written to set a non-exhaustive framework for shared understanding and discussion across disciplines and appeal to scientists and non-scientists alike. This appeal is made inclusively, inviting all stakeholders to engage in active dialogue about the appropriate context for using CRISPR and other gene-editing technologies in humans. It provides policy analysis and recommendations for assuring the most inclusive, equitable, and ethically sound use of CRISPR in humans, concerning its positive potential to treat mental conditions like depression, schizophrenia, Alzheimer’s disease, autism, and the potential to induce other cognitive enhancements.

Download or read book CRISPR Genome Surgery in Stem Cells and Disease Tissues written by Stephen H. Tsang and published by Academic Press. This book was released on 2021-10-20 with total page 138 pages. Available in PDF, EPUB and Kindle. Book 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

Download or read book Reprogramming the Genome Applications of CRISPR Cas in non mammalian systems part B written by and published by Academic Press. This book was released on 2021-04-29 with total page 200 pages. Available in PDF, EPUB and Kindle. Book 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

Download or read book Gene Editing in Plants written by and published by Academic Press. This book was released on 2017-07-14 with total page 266 pages. Available in PDF, EPUB and Kindle. Book excerpt: Gene Editing in Plants, Volume 149 aims to provide the reader with an up-to-date survey of cutting-edge research with gene editing tools and an overview of the implications of this research on the nutritional quality of fruits, vegetables and grains. New chapters in the updated volume include topics relating to Genome Engineering and Agriculture: Opportunities and Challenges, the Use of CRISPR/Cas9 for Crop Improvement in Maize and Soybean, the Use of Zinc-Finger Nucleases for Crop Improvement, Gene Editing in Polyploid Crops: Wheat, Camelina, Canola, Potato, Cotton, Peanut, Sugar Cane, and Citrus, and Gene Editing With TALEN and CRISPR/Cas in Rice. This ongoing serial contain contributions from leading scientists and researchers in the field of gene editing in plants who describe the results of their own research in this rapidly expanding area of science. - Shows the importance of revolutionary gene editing technology on plant biology research and its application to agricultural production - Provides insight into what may lie ahead in this rapidly expanding area of plant research and development - Contains contributions from major leaders in the field of plant gene editing

Download or read book Engineering Efficient and Safe in Situ Genome Regulation Via CRISPR Cas9 for Enabling Gene Therapies written by Ana Maria Moreno and published by . This book was released on 2019 with total page 175 pages. Available in PDF, EPUB and Kindle. Book 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 concomitant prevention of secondary cone loss. Furthermore, we benchmarked our results from Nrl knockdown with those from in vivo Nrl knockout via gene editing. Taken together, our AAV-CRISPR-Cas9 platform for in vivo epigenome engineering enables a robust approach to target disease in a genomically scarless and potentially reversible manner. Additionally, this is the first time that the utility of AAV-KRAB-dCas9 mediated in situ gene repression in the context of gene therapy was demonstrated (Moreno et al., WIREs Systems Biology and Medicine, 2017; Moreno et al., Molecular Therapy, 2018). Next, I focused on addressing, arguably the most important hurdle for CRISPR-Cas based gene therapies, which is the interaction of these non-host derived systems with the adaptive immune system which can lead to neutralization by circulating antibodies and clearance of treated cells by cytotoxic T-lymphocytes. To address this issue, I proposed a new approach: sequential use of orthologous proteins that are orthogonal in immune space. This would, in principle, allow for repeated treatments by thus chosen orthologs without reduced efficacy due to lack of immune cross-reactivity among the proteins. To explore and validate this concept we chose 284 DNA targeting and 84 RNA targeting CRISPR effectors (including Cas9, Cpf1/Cas12a, and Cas13a, b, and c), and 167 Adeno-associated virus (AAV) capsid protein orthologs and developed a pipeline to compare total sequence similarity as well as predicted binding to class I and class II Major Histocompatibility Complex (MHC) proteins. Our MHC binding predictions revealed wide diversity among the set of DNA-targeting Cas orthologs, with 79% of pairs predicted not to elicit cross-reacting immune responses, while no global immune orthogonality among AAV serotypes was observed. We validated the computationally predicted immune orthogonality among three important Cas9 orthologs, from S. pyogenes, S. aureus, and C. jejuni observing cross-reacting antibodies against AAV but not Cas9 orthologs in sera from immunized mice. Finally, to demonstrate the efficacy of multiple dosing with immune orthogonal orthologs, we delivered AAV-Cas9 targeting PCSK9 into BALB/c mice previously immunized against the AAV vector and/or the Cas9 payload, demonstrating that editing efficiency is compromised by immune recognition of either the AAV or Cas9, but, importantly, this effect is abrogated when using immune orthogonal orthologs. Moving forward, we anticipate this framework can be applied to prescribe sequential transient regimens of immune orthogonal protein therapeutics to circumvent pre-existing or induced immunity, and eventually, to rationally engineer immune orthogonality among protein orthologs. (Moreno, Palmer et al., Nature Biomedical Engineering, in press, 2019). Lastly, I then proceeded to integrate the advances accomplished in the previous chapters to enable pain management via in situ genome repression. In the US and worldwide, pain is a leading cause of disability, which leads to a diminished quality of life. Patients have come to routinely expect pharmacological management, with the prevalent aggressive approach for managing pain states being based on opiates. While the utility of opiates has made them a mainstay of pain management, there are at least four key reasons supporting the need for new and alternative pain therapeutics: limited efficacy, abuse potential, tolerance after continued exposure, and an enhancement of post-wound pain states. Despite decades of research, broad-acting, longer-lived, non-addictive, and effective drugs for chronic pain remain elusive. Notably, genetic studies have correlated a hereditary loss-of-function mutation in a human Na+ channel isoform--NaV1.7--with a rare genetic disorder, Congenital Insensitivity to Pain (CIP), which leads to insensitivity to pain without other neurodevelopmental alterations. While an excellent target, the creation of blockers for this site has not led yet to an efficient and safe drug, due to their lack of specificity, leading to unwanted side-effects. Taking advantage of this druggable target in the human genome, the aim of this work was to develop a novel therapeutic regiment via in situ NaV1.7 repression to regulate the development and maintenance of impending chronic pain states. In this regard, I demonstrated robust in vitro repression using two epigenome engineering tools (KRAB-CRISPR-Cas9 and KRAB-Zinc-Fingers) with ~71% and ~88% repression respectively. To enable pain management, I injected mice intrathecally using the constructs with the highest in vitro efficacy and demonstrated robust NaV1.7 repression with a significant improvement in pain response in a carrageenan inflammatory pain model. I demonstrated a 133% improvement in paw withdrawal latency as compared to a negative control (mCherry) and 62% improvement over the positive control (Gabapentin, 100 mg/kg). Taken together, the use of these in situ engineering approaches could thus represent a viable replacement for opioids and a potential therapeutic approach that is tunable and reversible (Moreno et al., in preparation). Together, the advances in these bodies of work, which demonstrate efficacious in vivo delivery and gene editing/regulation is a significant step toward their implementation for gene therapeutic applications.

Download or read book Genome Editing in Neurosciences written by Rudolf Jaenisch and published by Springer. This book was released on 2017-09-14 with total page 129 pages. Available in PDF, EPUB and Kindle. Book excerpt: This book is open access under a CC BY 4.0 license. CRISPR-Cas9 is a rapid, efficient, versatile and relatively cheap method for dissecting the molecular pathways that are the basis of life, as well as for investigating and potentially rectifying faults in these pathways that result in disease. This book reviews how CRISPR-Cas9 and other genome editing techniques are advancing our understanding of development and function in the nervous system, uncovering the molecular causes of neurological disorders and providing tools for gene therapy.

Download or read book Precision Medicine CRISPR and Genome Engineering written by Stephen H. Tsang and published by Springer. This book was released on 2017-11-11 with total page 180 pages. Available in PDF, EPUB and Kindle. Book excerpt: This book presents descriptive overviews of gene editing strategies across multiple species while also offering in-depth insight on complex cases of application in the field of tissue engineering and regenerative medicine. Chapters feature contributions from leaders in stem cell therapy and biology, providing a comprehensive view of the application of gene therapy in numerous fields with an emphasis on ophthalmology, stem cells, and agriculture. The book also highlights recent major technological advances, including ZFN, TALEN, and CRISPR. Precision Medicine, CRISPR, and Genome Engineering is part of the highly successful Advances in Experimental Medicine and Biology series. It is an indispensable resource for researchers and students in genetics as well as clinicians.

Download or read book Genome Editing in Neurosciences written by Rudolf Jaenisch and published by . This book was released on 2020-10-08 with total page 128 pages. Available in PDF, EPUB and Kindle. Book excerpt: Innovations in molecular biology are allowing neuroscientists to study the brain with unprecedented resolution, from the level of single molecules to integrated gene circuits. Chief among these innovations is the CRISPR-Cas genome editing technology, which has the precision and scalability to tackle the complexity of the brain. This Colloque Médecine et Recherche has brought together experts from around the world that are applying genome editing to address important challenges in neuroscience, including basic biology in model organisms that has the power to reveal systems-level insight into how the nervous system develops and functions as well as research focused on understanding and treating human neurological disorders. This work was published by Saint Philip Street Press pursuant to a Creative Commons license permitting commercial use. All rights not granted by the work's license are retained by the author or authors.

Download or read book CRISPR in Animals and Animal Models written by and published by Academic Press. This book was released on 2017-11-10 with total page 150 pages. Available in PDF, EPUB and Kindle. Book 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

Download or read book CRISPR Genome Editing and Engineering And Related Issues written by Barbara Wexler and published by Gale, Cengage Learning. This book was released on 2018-11-16 with total page 68 pages. Available in PDF, EPUB and Kindle. Book excerpt: eBook content that offers a clear and comprehensive introduction to CRISPR and related topics. Entries include foundational concepts, key scientific figures and historical themes, ethical issues , and advances in the science.