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Mutation Specific Gene Editing For Blood Disorders
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Book Synopsis Mutation-Specific Gene Editing for Blood Disorders by : Carsten Werner Lederer
Download or read book Mutation-Specific Gene Editing for Blood Disorders written by Carsten Werner Lederer and published by Frontiers Media SA. This book was released on 2021-11-25 with total page 218 pages. Available in PDF, EPUB and Kindle. Book excerpt: Dr. Miccio holds patents related to viral gene delivery vectors. The Topic Editors acknowledge the use of image material from kindpng.com and from Crystal and Annie Spratt on unsplash.com.
Book Synopsis Heritable Human Genome Editing by : The Royal Society
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.
Book Synopsis Gene Therapy for Cancer by : Kelly K. Hunt
Download or read book Gene Therapy for Cancer written by Kelly K. Hunt and published by Springer Science & Business Media. This book was released on 2007-10-26 with total page 469 pages. Available in PDF, EPUB and Kindle. Book excerpt: The three sections of this volume present currently available cancer gene therapy techniques. Part I describes the various aspects of gene delivery. In Part II, the contributors discuss strategies and targets for the treatment of cancer. Finally, in Part III, experts discuss the difficulties inherent in bringing gene therapy treatment for cancer to the clinic. This book will prove valuable as the volume of preclinical and clinical data continues to increase.
Author :National Academies of Sciences, Engineering, and Medicine Publisher :National Academies Press ISBN 13 :0309452880 Total Pages :329 pages Book Rating :4.3/5 (94 download)
Book Synopsis Human Genome Editing by : National Academies of Sciences, Engineering, and Medicine
Download or read book Human Genome Editing written by National Academies of Sciences, Engineering, and Medicine and published by National Academies Press. This book was released on 2017-08-13 with total page 329 pages. Available in PDF, EPUB and Kindle. Book 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.
Book Synopsis Translating Gene Therapy to the Clinic by : Jeffrey Laurence
Download or read book Translating Gene Therapy to the Clinic written by Jeffrey Laurence and published by Academic Press. This book was released on 2014-11-14 with total page 347 pages. Available in PDF, EPUB and Kindle. Book excerpt: Translating Gene Therapy to the Clinic, edited by Dr. Jeffrey Laurence and Michael Franklin, follows the recent, much-lauded special issue of Translational Research in emphasizing clinical milestones and critical barriers to further progress in the clinic. This comprehensive text provides a background for understanding the techniques involved in human gene therapy trials, and expands upon the disease-specific situations in which these new approaches currently have the greatest therapeutic application or potential, and those areas most in need of future research. It emphasizes methods, tools, and experimental approaches used by leaders in the field of translational gene therapy. The book promotes cross-disciplinary communication between the sub-specialties of medicine, and remains unified in theme. Presents impactful and widely supported research across the spectrum of science, method, implementation and clinical application Offers disease-based coverage from expert clinician-scientists, covering everything from arthritis to congestive heart failure, as it details specific progress and barriers for current translational use Provides key background information from immune response through genome engineering and gene transfer, relevant information for practicing clinicians contemplating enrolling patients in gene therapy trials
Book Synopsis Gene Therapy, An Issue of Hematology/Oncology Clinics of North America, E-Book by : Daniel E. Bauer
Download or read book Gene Therapy, An Issue of Hematology/Oncology Clinics of North America, E-Book written by Daniel E. Bauer and published by Elsevier Health Sciences. This book was released on 2017-09-27 with total page pages. Available in PDF, EPUB and Kindle. Book excerpt: This issue of Hematology/Oncology Clinics will focus on Gene Therapy. Topics include, but are not limited to Historical Perspective and Current Renaissance, Integrating Vectors, Nonintegrating Vectors, Gene Editing, Conditioning Therapies for Autologous HSCT, Approaches to Immunodeficiency, Approaches to Hemoglobinopathy, Approaches to Hemophilia, Hematopoietic Gene Therapies for Neurologic and Metabolic Disease, Gene Therapy Approaches to HIV and other Infectious Diseases, HSC Approaches to Cancer, and Gene Modified T Cell Therapies for Cancer.
Book Synopsis CRISPR-Cas Systems by : Rodolphe Barrangou
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.
Book Synopsis Immunopharmacology by : Manzoor M. Khan
Download or read book Immunopharmacology written by Manzoor M. Khan and published by Springer Science & Business Media. This book was released on 2008-12-19 with total page 275 pages. Available in PDF, EPUB and Kindle. Book excerpt: During the past decades, with the introduction of the recombinant DNA, hybridoma and transgenic technologies there has been an exponential evolution in understanding the pathogenesis, diagnosis and treatment of a large number of human diseases. The technologies are evident with the development of cytokines and monoclonal antibodies as therapeutic agents and the techniques used in gene therapy. Immunopharmacology is that area of biomedical sciences where immunology, pharmacology and pathology overlap. It concerns the pharmacological approach to the immune response in physiological as well as pathological events. This goals and objectives of this textbook are to emphasize the developments in immunology and pharmacology as they relate to the modulation of immune response. The information includes the pharmacology of cytokines, monoclonal antibodies, mechanism of action of immune-suppressive agents and their relevance in tissue transplantation, therapeutic strategies for the treatment of AIDS and the techniques employed in gene therapy. The book is intended for health care professional students and graduate students in pharmacology and immunology.
Book Synopsis Somatic Gene Therapy by : P.L. Chang
Download or read book Somatic Gene Therapy written by P.L. Chang and published by CRC Press. This book was released on 2018-01-17 with total page 242 pages. Available in PDF, EPUB and Kindle. Book excerpt: As human gene therapy becomes a clinical reality, a new era in medicine dawns. Novel and innovative developments in molecular genetics now provide opportunities to treat the genetic bases of diseases often untreatable before. Somatic Gene Therapy documents these historical clinical trials, reviews current advances in the field, evaluates the use of the many different cell types and organs amenable to gene transfer, and examines the prospects of various exciting strategies for gene therapy.
Book Synopsis Genome Editing in Neurosciences by : Rudolf Jaenisch
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 Gene Editing written by Yuan-Chuan Chen and published by BoD – Books on Demand. This book was released on 2019-05-29 with total page 110 pages. Available in PDF, EPUB and Kindle. Book excerpt: Gene-editing technologies (e.g., ZFNs, TALENs, and CRISPRs/Cas9) have been extensively used as tools in basic research. They are further applied in manufacturing agricultural products, food, industrial products, medicinal products, etc. Particularly, the discovery of medicinal products using gene-editing technologies will open a new era for human therapeutics. Though there are still many technical and ethical challenges ahead of us, more and more products based on gene-editing technologies have been approved for marketing. These technologies are promising for multiple applications. Their development and implications should be explored in the broadest context possible. Future research directions should also be highlighted. In this book, the applications, perspectives, and challenges of gene-editing technologies are significantly demonstrated and discussed.
Book Synopsis Renaissance Of Sickle Cell Disease Research In The Genome Era by : Betty Pace
Download or read book Renaissance Of Sickle Cell Disease Research In The Genome Era written by Betty Pace and published by World Scientific. This book was released on 2007-01-24 with total page 394 pages. Available in PDF, EPUB and Kindle. Book excerpt: The Human Genome Project has spawned a Renaissance of research faced with the daunting expectation of personalized medicine for individuals with sickle cell disease in the Genome Era. This book offers a comprehensive and timeless account of emerging concepts in clinical and basic science research, and community concerns of health disparity to educate professionals, students and the general public about meeting this challenging expectation. Contributions from physicians, research scientists, scientific administrators and community workers make Renaissance of Sickle Cell Disease Research in the Genome Era unique among the catalogue of books on this genetic disorder.Part 1 offers detailed review of the National Heart Lung and Blood Institute's leadership role in funding sickle cell research, as well as developing progressive research initiatives and the predicted impact of the Human Genome Project. Part 2 gives an account of several clinical research perspectives based on the Cooperative Study of Sickle Cell Disease. These include recommendations for newborn screening, pain management, stroke, transfusion therapy and pediatric and adult healthcare. Part 3 offers novel insights into basic science research progress and the impact of the Human Genome Project on the direction of hemoglobinopathy research, including hemoglobin switching, bone marrow transplantation and gene therapy. Part 4 engages the reader in a culture-based discussion of the stigma attached to sickle cell disease in the African American community and the apprehensions about genetic research in this community. It concludes with a global perspective on sickle cell disease from African, European and American experiences. For readers seeking a definitive account of sickle cell disease appropriate for students, researchers and community workers, this collaborative effort is an ideal textbook./a
Book Synopsis Thomas' Hematopoietic Cell Transplantation by : Stephen J. Forman
Download or read book Thomas' Hematopoietic Cell Transplantation written by Stephen J. Forman and published by John Wiley & Sons. This book was released on 2015-12-14 with total page 1416 pages. Available in PDF, EPUB and Kindle. Book excerpt: Fully revised for the fifth edition, this outstanding reference on bone marrow transplantation is an essential, field-leading resource. Extensive coverage of the field, from the scientific basis for stem-cell transplantation to the future direction of research Combines the knowledge and expertise of over 170 international specialists across 106 chapters Includes new chapters addressing basic science experiments in stem-cell biology, immunology, and tolerance Contains expanded content on the benefits and challenges of transplantation, and analysis of the impact of new therapies to help clinical decision-making Includes a fully searchable Wiley Digital Edition with downloadable figures, linked references, and more References for this new edition are online only, accessible via the Wiley Digital Edition code printed inside the front cover or at www.wiley.com/go/forman/hematopoietic.
Book Synopsis Pharmaceutical Biotechnology by : Daan J. A. Crommelin
Download or read book Pharmaceutical Biotechnology written by Daan J. A. Crommelin and published by CRC Press. This book was released on 2002-11-14 with total page 456 pages. Available in PDF, EPUB and Kindle. Book excerpt: The field of pharmaceutical biotechnology is evolving rapidly. A whole new arsenal of protein pharmaceuticals is being produced by recombinant techniques for cancer, viral infections, cardiovascular and hereditary disorders, and other diseases. In addition, scientists are confronted with new technologies such as polymerase chain reactions, combinatorial chemistry and gene therapy. This introductory textbook provides extensive coverage of both the basic science and the applications of biotechnology-produced pharmaceuticals, with special emphasis on their clinical use. Pharmaceutical Biotechnology serves as a complete one-stop source for undergraduate pharmacists, and it is valuable for researchers and professionals in the pharmaceutical industry as well.
Download or read book Middlesex written by Jeffrey Eugenides and published by Vintage Canada. This book was released on 2011-07-18 with total page 546 pages. Available in PDF, EPUB and Kindle. Book excerpt: Spanning eight decades and chronicling the wild ride of a Greek-American family through the vicissitudes of the twentieth century, Jeffrey Eugenides’ witty, exuberant novel on one level tells a traditional story about three generations of a fantastic, absurd, lovable immigrant family -- blessed and cursed with generous doses of tragedy and high comedy. But there’s a provocative twist. Cal, the narrator -- also Callie -- is a hermaphrodite. And the explanation for this takes us spooling back in time, through a breathtaking review of the twentieth century, to 1922, when the Turks sacked Smyrna and Callie’s grandparents fled for their lives. Back to a tiny village in Asia Minor where two lovers, and one rare genetic mutation, set our narrator’s life in motion. Middlesex is a grand, utterly original fable of crossed bloodlines, the intricacies of gender, and the deep, untidy promptings of desire. It’s a brilliant exploration of divided people, divided families, divided cities and nations -- the connected halves that make up ourselves and our world.
Book Synopsis Modulating Gene Expression by : Aditi Singh
Download or read book Modulating Gene Expression written by Aditi Singh and published by BoD – Books on Demand. This book was released on 2019-05-29 with total page 136 pages. Available in PDF, EPUB and Kindle. Book excerpt: RNA interference (RNAi) is a widely used technology for gene silencing and has become a key tool in a myriad of research and lead discoveries. In recent years, the mechanism of RNAi agents has been well investigated, and the technique has been optimized for better effectiveness and safety. On the other hand, the clustered regularly interspaced short palindromic repeats (CRISPR)-associated Cas9/gRNA system is a recent, novel, targeted genome-editing technique derived from the bacterial immune system. Recent advances in gene-editing research and technologies have enabled the CRISPR Cas9 system to become a popular tool for sequence-specific gene editing to correct and modify eukaryotic systems. In this book, we will focus on the mechanisms, applications, regulations (their pros and cons), and various ways in which RNAi-based methods and CRIPSR-Cas9 technology have stimulated the modulation of gene expression, thereby making them a promising therapeutic tool to treat and prevent complex diseases and disorders.
Book Synopsis Site-Specific Gene Correction of Hematopoietic Stem Cells for Sickle Cell Disease by : Megan D. Hoban
Download or read book Site-Specific Gene Correction of Hematopoietic Stem Cells for Sickle Cell Disease written by Megan D. Hoban and published by . This book was released on 2015 with total page 123 pages. Available in PDF, EPUB and Kindle. Book excerpt: Sickle cell disease is one of the most common monogenic diseases in the world and, since its observation over 100 years ago, has become one of the most well-studied genetic diseases. The causal single point mutation in the beta-globin gene leads to the formation of hemoglobin polymers in the effected red blood cells under low oxygen conditions. While oral therapies are available to relieve some of the underlying symptoms, the only currently available cure is a hematopoietic stem cell transplant from a matched donor. However, most patients do not have an available donor and transplants carry the risk of rejection and immune complications. Therefore, gene therapy for sickle cell disease using a patient's own hematopoietic stem cells offers the potential for a more viable treatment. The use of lentiviral vectors for gene therapy for the hemoglobinopathies has an established clinical course and several groups are currently in the midst of early trials. However, integrating vectors carry with them the risk for insertional oncogenesis as well as silencing or irregular expression of the transgene. Thus, targeted correction of the causal mutation would be an ideal approach for the treatment of sickle cell disease. Here we present data using targeted endonucleases in combination with a corrective donor template to induce targeted double-strand breaks in exon 1 of beta-globin and the subsequent repair of the break by homology directed repair in both cell lines as well as hematopoietic stem and progenitor cells. We describe the development of the reagents necessary to achieve this targeted correction, the optimization of delivery platforms for the nucleases as well as the donor template, and the refinement of the protocol for the treatment of CD34+ hematopoietic stem and progenitor cells. In addition, we demonstrate the ability of these nuclease and donor-modified cells to engraft an immunocompromised murine model and differentiate into multiple hematopoietic lineages. Finally, we show the correction of bone marrow hematopoietic stem and progenitor cells from sickle cell disease patients, resulting in the production of wild-type, adult hemoglobin tetramers. These data represent the foundational evidence for the site-specific gene correction of hematopoietic stem cells for sickle cell disease. We include a thorough description of the achievements made toward this aim as well as the remaining objectives and hurdles that face the genome editing field.
