Curing Genetic Diseases Through Genome Reprogramming

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Curing Genetic Diseases through Genome Reprogramming

Author :
Publisher : Academic Press
ISBN 13 : 0323853021
Total Pages : 560 pages
Book Rating : 4.3/5 (238 download)

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Book Synopsis Curing Genetic Diseases through Genome Reprogramming by :

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

Reprogramming the Genome: CRISPR-Cas-based Human Disease Therapy

Author :
Publisher : Academic Press
ISBN 13 : 0323853242
Total Pages : 394 pages
Book Rating : 4.3/5 (238 download)

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Book Synopsis Reprogramming the Genome: CRISPR-Cas-based Human Disease Therapy by :

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

Gene Therapy

Author : Joseph Panno
Publisher : Infobase Publishing
ISBN 13 : 0816067341
Total Pages : 193 pages
Book Rating : 4.8/5 (16 download)

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Book Synopsis Gene Therapy by : Joseph Panno

Download or read book Gene Therapy written by Joseph Panno and published by Infobase Publishing. This book was released on 2005 with total page 193 pages. Available in PDF, EPUB and Kindle. Book excerpt: Many diseases are caused by a simple point mutation.

Gene Editing in Plants

Author :
Publisher : Academic Press
ISBN 13 : 0128117443
Total Pages : 266 pages
Book Rating : 4.1/5 (281 download)

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Book Synopsis Gene Editing in Plants by :

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

Heritable Human Genome Editing

Author : The Royal Society
Publisher : National Academies Press
ISBN 13 : 0309671132
Total Pages : 239 pages
Book Rating : 4.3/5 (96 download)

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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.

CRISPR-Cas Systems

Author : Rodolphe Barrangou
Publisher : Springer Science & Business Media
ISBN 13 : 364234657X
Total Pages : 300 pages
Book Rating : 4.6/5 (423 download)

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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.

Genome Editing in Neurosciences

Author : Rudolf Jaenisch
Publisher :
ISBN 13 : 9781013268564
Total Pages : 128 pages
Book Rating : 4.2/5 (685 download)

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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.

Human Genome Editing

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)

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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.

CRISPR in Animals and Animal Models

Author :
Publisher : Academic Press
ISBN 13 : 0128125071
Total Pages : 150 pages
Book Rating : 4.1/5 (281 download)

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Book Synopsis CRISPR in Animals and Animal Models by :

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

Hacking Darwin

Author : Jamie Metzl
Publisher : Sourcebooks, Inc.
ISBN 13 : 1492670103
Total Pages : 253 pages
Book Rating : 4.4/5 (926 download)

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Book Synopsis Hacking Darwin by : Jamie Metzl

Download or read book Hacking Darwin written by Jamie Metzl and published by Sourcebooks, Inc.. This book was released on 2019-04-23 with total page 253 pages. Available in PDF, EPUB and Kindle. Book excerpt: "A gifted and thoughtful writer, Metzl brings us to the frontiers of biology and technology, and reveals a world full of promise and peril." — Siddhartha Mukherjee MD, New York Times bestselling author of The Emperor of All Maladies and The Gene A groundbreaking exploration of genetic engineering and its impact on the future of our species from leading geopolitical expert and technology futurist, Jamie Metzl. At the dawn of the genetics revolution, our DNA is becoming as readable, writable, and hackable as our information technology. But as humanity starts retooling our own genetic code, the choices we make today will be the difference between realizing breathtaking advances in human well-being and descending into a dangerous and potentially deadly genetic arms race. Enter the laboratories where scientists are turning science fiction into reality. In this captivating and thought-provoking nonfiction science book, Jamie Metzl delves into the ethical, scientific, political, and technological dimensions of genetic engineering, and shares how it will shape the course of human evolution. Cutting-edge insights into the field of genetic engineering and its implications for humanity's future Explores the transformative power of genetic technologies and their potential to reshape human life Examines the ethical considerations surrounding genetic engineering and the choices we face as a species Engaging narrative that delves into the scientific breakthroughs and real-world applications of genetic technologies Provides a balanced perspective on the promises and risks associated with genetic engineering Raises thought-provoking questions about the future of reproduction, human health, and our relationship with nature Drawing on his extensive background in genetics, national security, and foreign policy, Metzl paints a vivid picture of a world where advancements in technology empower us to take control of our own evolution, but also cautions against the pitfalls and ethical dilemmas that could arise if not properly managed. Hacking Darwin is a must-read for anyone interested in the intersection of science, technology, and humanity's future.

An Introduction to Ethical, Safety and Intellectual Property Rights Issues in Biotechnology

Author : Padma Nambisan
Publisher : Academic Press
ISBN 13 : 0128092513
Total Pages : 394 pages
Book Rating : 4.1/5 (28 download)

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Book Synopsis An Introduction to Ethical, Safety and Intellectual Property Rights Issues in Biotechnology by : Padma Nambisan

Download or read book An Introduction to Ethical, Safety and Intellectual Property Rights Issues in Biotechnology written by Padma Nambisan and published by Academic Press. This book was released on 2017-06-21 with total page 394 pages. Available in PDF, EPUB and Kindle. Book excerpt: An Introduction to Ethical, Safety and Intellectual Property Rights Issues in Biotechnology provides a comprehensive look at the biggest technologies that have revolutionized biology since the early 20th century, also discussing their impact on society. The book focuses on issues related to bioethics, biosafety and intellectual property rights, and is written in an easy-to-understand manner for graduate students and early career researchers interested in the opportunities and challenges associated with advances in biotechnology. Important topics covered include the Human Genome Project, human cloning, rDNA technology, the 3Rs and animal welfare, bioterrorism, human rights and genetic discrimination, good laboratory practices, good manufacturing practices, the protection of biological material and much more. Full of relevant case studies, practical examples, weblinks and resources for further reading, this book offers an essential and holistic look at the ways in which biotechnology has affected our global society. Provides a comprehensive look at the ethical, legal and social implications of biotechnology Discusses the global efforts made to resolve issues Incorporates numerous case studies to more clearly convey concepts and chart the development of guidelines and legislation regulating issues in biotechnology Takes a straightforward approach to highlight and discuss both the benefits and risks associated with the latest biotechnologies

Genomic Disorders

Author : James R. Lupski
Publisher : Springer Science & Business Media
ISBN 13 : 1597450391
Total Pages : 419 pages
Book Rating : 4.5/5 (974 download)

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Book Synopsis Genomic Disorders by : James R. Lupski

Download or read book Genomic Disorders written by James R. Lupski and published by Springer Science & Business Media. This book was released on 2007-11-10 with total page 419 pages. Available in PDF, EPUB and Kindle. Book excerpt: A grand summary and synthesis of the tremendous amount of data now available in the post genomic era on the structural features, architecture, and evolution of the human genome. The authors demonstrate how such architectural features may be important to both evolution and to explaining the susceptibility to those DNA rearrangements associated with disease. Technologies to assay for such structural variation of the human genome and to model genomic disorders in mice are also presented. Two appendices detail the genomic disorders, providing genomic features at the locus undergoing rearrangement, their clinical features, and frequency of detection.

CRISPR

Author : Rodolphe Barrangou
Publisher : John Wiley & Sons
ISBN 13 : 168367037X
Total Pages : 308 pages
Book Rating : 4.6/5 (836 download)

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Book Synopsis CRISPR by : Rodolphe Barrangou

Download or read book CRISPR written by Rodolphe Barrangou and published by John Wiley & Sons. This book was released on 2022-09-14 with total page 308 pages. Available in PDF, EPUB and Kindle. Book excerpt: “An excellent compendium of all things CRISPR from some of the leading minds in the field. With thorough coverage from every angle and beautifully detailed illustrations, this book is not to be missed!” Jennifer A. Doudna, Professor of Chemistry, Biochemistry & Molecular Biology, UC Berkeley; Founder, Innovative Genomics Institute; Nobel Laureate and coinventor of CRISPR technology “This journey through CRISPR biology and several of its breakthrough applications offers an exciting glimpse into one of the most beautiful and compelling fields in the life sciences.” David R. Liu, Director of the Merkin Institute at the Broad Institute of MIT and Harvard; Professor of Chemistry and Chemical Biology at Harvard University; coinventor of base editing and prime editing “A must read! The CRISPR topics, written by world-leading experts, span from the fascinating mechanistic underpinnings to the ingenious applications. One can read from start to finish or pick and choose themes. Either way, the book delivers utterly enjoyable learning!” Bonnie Bassler, Squibb Professor and Chair, Princeton University Department of Molecular Biology; Howard Hughes Medical Institute Investigator CRISPR-Cas systems have revolutionized the science of gene editing and their possible applications continue to expand, from basic research to potentially groundbreaking medical and commercial uses. Led by a distinguished team of editors, CRISPR: Biology and Applications explores the subject matter needed to delve into this fascinating area. Topics covered include: Classification and molecular mechanisms of CRISPR-Cas systems CRISPR-Cas evolution, regulation, expression, and function Uses for gene editing and modulation of gene expression CRISPR-based antimicrobials and phage resistance for medical and industrial purposes Written by internationally renowned authors, CRISPR: Biology and Applications serves as both an introductory guide for those new to the field and an authoritative reference for seasoned researchers whose work touches this evolving and headline- making science.

Introduction to Basics of Pharmacology and Toxicology

Author : Gerard Marshall Raj
Publisher : Springer Nature
ISBN 13 : 9813297794
Total Pages : 410 pages
Book Rating : 4.8/5 (132 download)

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Book Synopsis Introduction to Basics of Pharmacology and Toxicology by : Gerard Marshall Raj

Download or read book Introduction to Basics of Pharmacology and Toxicology written by Gerard Marshall Raj and published by Springer Nature. This book was released on 2019-11-16 with total page 410 pages. Available in PDF, EPUB and Kindle. Book excerpt: This book illustrates, in a comprehensive manner, the most crucial principles involved in pharmacology and allied sciences. The title begins by discussing the historical aspects of drug discovery, with up to date knowledge on Nobel Laureates in pharmacology and their significant discoveries. It then examines the general pharmacological principles - pharmacokinetics and pharmacodynamics, with in-depth information on drug transporters and interactions. In the remaining chapters, the book covers a definitive collection of topics containing essential information on the basic principles of pharmacology and how they are employed for the treatment of diseases. Readers will learn about special topics in pharmacology that are hard to find elsewhere, including issues related to environmental toxicology and the latest information on drug poisoning and treatment, analytical toxicology, toxicovigilance, and the use of molecular biology techniques in pharmacology. The book offers a valuable resource for researchers in the fields of pharmacology and toxicology, as well as students pursuing a degree in or with an interest in pharmacology.

Advanced Textbook On Gene Transfer, Gene Therapy And Genetic Pharmacology: Principles, Delivery And Pharmacological And Biomedical Applications Of Nucleotide-based Therapies

Download Advanced Textbook On Gene Transfer, Gene Therapy And Genetic Pharmacology: Principles, Delivery And Pharmacological And Biomedical Applications Of Nucleotide-based Therapies PDF Online Free

Author : Daniel Scherman
Publisher : World Scientific
ISBN 13 : 1783263148
Total Pages : 557 pages
Book Rating : 4.7/5 (832 download)

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Book Synopsis Advanced Textbook On Gene Transfer, Gene Therapy And Genetic Pharmacology: Principles, Delivery And Pharmacological And Biomedical Applications Of Nucleotide-based Therapies by : Daniel Scherman

Download or read book Advanced Textbook On Gene Transfer, Gene Therapy And Genetic Pharmacology: Principles, Delivery And Pharmacological And Biomedical Applications Of Nucleotide-based Therapies written by Daniel Scherman and published by World Scientific. This book was released on 2013-12-10 with total page 557 pages. Available in PDF, EPUB and Kindle. Book excerpt: This unique advanced textbook provides a clear and comprehensive description of the field of gene delivery, gene therapy and genetic pharmacology, with descriptions of the main gene transfer vectors and a set of selected therapeutic applications, along with safety considerations.The use of gene transfer is exponentially growing in the scientific and medical communities for day-to-day cell biology experiments and swift development of revolutionary gene therapy strategies. In this advanced textbook, more than 25 leading scientists, world-renowned in their respective fields, come together to provide a clear and comprehensive description of gene delivery, gene therapy and genetic pharmacology.This educational introduction to the main gene transfer vectors and selected therapeutic applications provides the background material needed to further explore the subject as well as relevant research literature. It will thus be invaluable to Master, PhD or MD students, post-doctoral scientists or medical doctors, as well as any scientist wishing to deliver a gene or synthetic nucleotide, or develop a gene therapy strategy. Furthermore, the textbook's simple and synthetic content will be of value to any reader interested in the biological and medical revolution derived from the elucidation of the human genome.

Epigenetics of Aging

Author : Trygve O. Tollefsbol
Publisher : Springer Science & Business Media
ISBN 13 : 1441906398
Total Pages : 462 pages
Book Rating : 4.4/5 (419 download)

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Book Synopsis Epigenetics of Aging by : Trygve O. Tollefsbol

Download or read book Epigenetics of Aging written by Trygve O. Tollefsbol and published by Springer Science & Business Media. This book was released on 2009-11-11 with total page 462 pages. Available in PDF, EPUB and Kindle. Book excerpt: Recent studies have indicated that epigenetic processes may play a major role in both cellular and organismal aging. These epigenetic processes include not only DNA methylation and histone modifications, but also extend to many other epigenetic mediators such as the polycomb group proteins, chromosomal position effects, and noncoding RNA. The topics of this book range from fundamental changes in DNA methylation in aging to the most recent research on intervention into epigenetic modifications to modulate the aging process. The major topics of epigenetics and aging covered in this book are: 1) DNA methylation and histone modifications in aging; 2) Other epigenetic processes and aging; 3) Impact of epigenetics on aging; 4) Epigenetics of age-related diseases; 5) Epigenetic interventions and aging: and 6) Future directions in epigenetic aging research. The most studied of epigenetic processes, DNA methylation, has been associated with cellular aging and aging of organisms for many years. It is now apparent that both global and gene-specific alterations occur not only in DNA methylation during aging, but also in several histone alterations. Many epigenetic alterations can have an impact on aging processes such as stem cell aging, control of telomerase, modifications of telomeres, and epigenetic drift can impact the aging process as evident in the recent studies of aging monozygotic twins. Numerous age-related diseases are affected by epigenetic mechanisms. For example, recent studies have shown that DNA methylation is altered in Alzheimer’s disease and autoimmunity. Other prevalent diseases that have been associated with age-related epigenetic changes include cancer and diabetes. Paternal age and epigenetic changes appear to have an effect on schizophrenia and epigenetic silencing has been associated with several of the progeroid syndromes of premature aging. Moreover, the impact of dietary or drug intervention into epigenetic processes as they affect normal aging or age-related diseases is becoming increasingly feasible.

A Guide to Human Gene Therapy

Author : Roland W. Herzog
Publisher : World Scientific
ISBN 13 : 9814280917
Total Pages : 415 pages
Book Rating : 4.8/5 (142 download)

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Book Synopsis A Guide to Human Gene Therapy by : Roland W. Herzog

Download or read book A Guide to Human Gene Therapy written by Roland W. Herzog and published by World Scientific. This book was released on 2010 with total page 415 pages. Available in PDF, EPUB and Kindle. Book excerpt: 1. Non-viral gene therapy / Sean M. Sullivan -- 2. Adenoviral vectors / Stuart A. Nicklin and Andrew H. Baker -- 3. Retroviral vectors and integration analysis / Cynthia C. Bartholomae [und weitere] -- 4. Lentiviral vectors / Janka Matrai, Marinee K.L. Chuah and Thierry VandenDriessche -- 5. Herpes simplex virus vectors / William F. Goins [und weitere] -- 6. Adeno-Associated Viral (AAV) vectors / Nicholas Muzyczka -- 7. Regulatory RNA in gene therapy / Alfred. S. Lewin -- 8. DNA integrating vectors (Transposon, Integrase) / Lauren E. Woodard and Michele P. Calos -- 9. Homologous recombination and targeted gene modification for gene therapy / Matthew Porteus -- 10. Gene switches for pre-clinical studies in gene therapy / Caroline Le Guiner [und weitere] -- 11. Gene therapy for central nervous system disorders / Deborah Young and Patricia A. Lawlor -- 12. Gene therapy of hemoglobinopathies / Angela E. Rivers and Arun Srivastava -- 13. Gene therapy for primary immunodeficiencies / Aisha Sauer, Barbara Cassani and Alessandro Aiuti -- 14. Gene therapy for hemophilia / David Markusic, Babak Moghimi and Roland Herzog -- 15. Gene therapy for obesity and diabetes / Sergei Zolotukhin and Clive H. Wasserfall -- 16. Gene therapy for Duchenne muscular dystrophy / Takashi Okada and Shin'ichi Takeda -- 17. Cancer gene therapy / Kirsten A.K. Weigel-Van Aken -- 18. Gene therapy for autoimmune disorders / Daniel F. Gaddy, Melanie A. Ruffner and Paul D. Robbins -- 19. Gene therapy for inherited metabolic storage diseases / Cathryn Mah -- 20. Retinal diseases / Shannon E. Boye, Sanford L. Boye and William W. Hauswirth -- 21. A brief guide to gene therapy treatments for pulmonary diseases / Ashley T. Martino, Christian Mueller and Terence R. Flotte -- 22. Cardiovascular disease / Darin J. Falk, Cathryn S. Mah and Barry J. Byrne