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Second Generation Cell and Gene-Based Therapies

Second Generation Cell and Gene-Based Therapies
  • Author : Alain Vertes,Nathan J. Dowden,Devyn Smith,Nasib Qureshi
  • Publisher :Unknown
  • Release Date :2020-02-07
  • Total pages :846
  • ISBN : 9780128120330
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Summary : Second Generation Cell and Gene-Based Therapies: Biological Advances, Clinical Outcomes, and Strategies for Capitalisation serves as the only volume to the market to bridge basic science, clinical therapy, technology development, and business in the field of cellular therapy/cytotherapy. After more than two decades of painstaking fundamental research, the concept of therapeutic cells (stem cells, genes, etc.), beyond the concept of vaccines, is reaching clinical trial, with mounting confidence in the safety and efficacy of these products. Nonetheless, numerous incremental technical advances remain to be achieved. Thus, this volume highlights the possible R&D paths, which will ultimately facilitate clinical delivery of cutting edge curative products. The next waves of innovation are reviewed in depth for hematopoietic stem cells, mesenchymal stem cells, tissue engineering, CAR-T cells, and cells of the immune system, as well as for enabling technologies such as gene and genome editing. Additionally, deep dives in product fundamentals, history of science, pathobiology of diseases, scientific and technological bases, and financing and technology adoption constraints are taken to unravel what will shape the cytotherapy industry to the horizon 2025 and beyond. The outcome is not simply a scientific book, but a global perspective on the nascent field combining science, business, and strategic fundamentals. Helps readers learn about the most current trends in cell-based therapy, their overall effectiveness from a clinical prospective, and how the industry is moving therapies forward for capitalization "Perspectives" section at the end of each chapter summarizes key learnings, hypotheses, and objectives highlighted and combines scientific and business insights Edited and authored by scientists representing both basic and clinical research and industry, presenting a complete story of the current state and future promise of cellular therapies

Stem Cell and Gene Therapy for Cardiovascular Disease

Stem Cell and Gene Therapy for Cardiovascular Disease
  • Author : Emerson C. Perin,Leslie W. Miller,Doris Taylor,James T. Willerson
  • Publisher :Unknown
  • Release Date :2015-08-21
  • Total pages :538
  • ISBN : 9780128018637
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Summary : Stem Cell and Gene Therapy for Cardiovascular Disease is a state-of-the-art reference that combines, in one place, the breadth and depth of information available on the topic. As stem cell and gene therapies are the most cutting-edge therapies currently available for patients with heart failure, each section of the book provides information on medical trials from contributors and specialists from around the world, including not only what has been completed, but also what is planned for future research and trials. Cardiology researchers, basic science clinicians, fellows, residents, students, and industry professionals will find this book an invaluable resource for further study on the topic. Provides information on stem and gene therapy medical trials from contributors and specialists around the world, including not only what has been completed, but also what is planned for future research and trials Presents topics that can be applied to allogeneic cells, mesenchymal cells, gene therapy, cardiomyoctyes, iPS cells, MAPC's, and organogenesis Covers the three areas with the greatest clinical trials to date: chronic limb ischemia, chronic angina, and acute MI Covers the prevailing opinions on how to harness the body’s natural repair mechanisms Ideal resource for cardiology researchers, basic science clinicians, fellows, residents, students, and industry professionals

Stem Cell and Gene-Based Therapy

Stem Cell and Gene-Based Therapy
  • Author : Alexander Battler,Jonathan Leor
  • Publisher :Unknown
  • Release Date :2007-06-26
  • Total pages :422
  • ISBN : 184628676X
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Summary : Regenerative medicine – stem cell and gene-based therapy – offers a new approach for restoring function of damaged organs and tissues. This is the first book to cover the major new aspects and field of regenerative medicine. This title is therefore a timely addition to the literature. It brings together the major approaches to regenerative medicine in one text, which ensures that techniques learnt in one discipline are disseminated across other areas of medicine.

Cell Therapy

Cell Therapy
  • Author : Adrian Gee
  • Publisher :Unknown
  • Release Date :2009-09-18
  • Total pages :256
  • ISBN : 0387895841
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Summary : Cell Therapy: cGMP Facilities and Manufacturing is the source for a complete discussion of facility design and operation with practical approaches to a variety of day-to-day activities, such as staff training and competency, cleaning procedures, and environmental monitoring. This in-depth book also includes detailed reviews of quality, the framework of regulations, and professional standards. It meets a previously unmet need for a thorough facility-focused resource, Cell Therapy: cGMP Facilities and Manufacturing will be an important addition to the cell therapy professional’s library. Additional topics in Cell Therapy: cGMP Facilities and Manufacturing...Standard operating procedures - Supply management - Facility equipment - Product manufacturing, review, release and administration - Facility master file.

Gene Therapy of Cancer

Gene Therapy of Cancer
  • Author : Stanton L. Gerson,Edmund C. Lattime
  • Publisher :Unknown
  • Release Date :2002-04-04
  • Total pages :534
  • ISBN : 0080491367
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Summary : The Second Edition of Gene Therapy of Cancer provides crucial updates on the basic science and ongoing research in this field, examining the state of the art technology in gene therapy and its therapeutic applications to the treatment of cancer. The clinical chapters are improved to include new areas of research and more successful trials. Chapters emphasize the scientific basis of gene therapy using immune, oncogene, antisense, pro-drug activating, and drug resistance gene targets, while other chapters discuss therapeutic approaches and clinical applications. This book is a valuable reference for anyone needing to stay abreast of the latest advances in gene therapy treatment for cancer. Key Features * Provides in-depth description of targeted systems and treatment strategies * Explains the underlying cancer biology necessary for understanding a given therapeutic approach * Extensively covers immune therapeutics of vaccines, cytokines, and peptide-induced responses * Presents translational focus with emphasis on requirements for clinical implementation * Incorporates detailed illustrations of vectors and therapeutic approaches ideal for classroom presentations and general reference

Braunwald's Heart Disease E-Book

Braunwald's Heart Disease E-Book
  • Author : Douglas L. Mann,Douglas P. Zipes,Peter Libby,Robert O. Bonow
  • Publisher :Unknown
  • Release Date :2014-07-30
  • Total pages :2136
  • ISBN : 9780323290647
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Summary : Ideal for cardiologists who need to keep abreast of rapidly changing scientific foundations, clinical research results, and evidence-based medicine, Braunwald’s Heart Disease is your indispensable source for definitive, state-of-the-art answers on every aspect of contemporary cardiology, helping you apply the most recent knowledge in personalized medicine, imaging techniques, pharmacology, interventional cardiology, electrophysiology, and much more! Practice with confidence and overcome your toughest challenges with advice from the top minds in cardiology today, who synthesize the entire state of current knowledge and summarize all of the most recent ACC/AHA practice guidelines. Locate the answers you need fast thanks to a user-friendly, full-color design with more than 1,200 color illustrations. Learn from leading international experts, including 53 new authors. Explore brand-new chapters, such as Principles of Cardiovascular Genetics and Biomarkers, Proteomics, Metabolomics, and Personalized Medicine. Access new and updated guidelines covering Diseases of the Aorta, Peripheral Artery Diseases, Diabetes and the Cardiovascular System, Heart Failure, and Valvular Heart Disease. Stay abreast of the latest diagnostic and imaging techniques and modalities, such as three-dimensional echocardiography, speckle tracking, tissue Doppler, computed tomography, and cardiac magnetic resonance imaging. Consult this title on your favorite e-reader, conduct rapid searches, and adjust font sizes for optimal readability.

Gene and Cell Therapy

Gene and Cell Therapy
  • Author : Nancy Smyth Templeton
  • Publisher :Unknown
  • Release Date :2008-10-06
  • Total pages :1120
  • ISBN : 084938799X
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Summary : Since the publication of the second edition of this book in 2004, gene therapy and cell therapy clinical trials have yielded some remarkable successes and some disappointing failures. Now in its third edition, Gene and Cell Therapy: Therapeutic Mechanisms and Strategies assembles many of the new technical advances in gene delivery, clinical applications, and new approaches to the regulation and modification of gene expression. New Topics Covered in this Edition: Gene and Cell Therapies for Diabetes and Cardiovascular Diseases Clinical Trials Human Embryonic Stem Cells Tissue Engineering Combined with Cell Therapies Novel Polymers Relevant Nanotechnologies SiRNA Therapeutic Strategies Dendrimer Technologies Comprised of contributions from international experts, this book begins with a discussion of delivery systems and therapeutic strategies, exploring retroviral vectors and adenovirus vectors, as well as other therapeutic strategies. The middle section focuses on gene expression and detection, followed by an examination of various therapeutic strategies for individual diseases, including hematopoietic disorders, cardiovascular conditions, cancer, diabetes, cystic fibrosis, neurological disorders, and childhood-onset blindness. The final section discusses recent clinical trials and regulatory issues surrounding the new technology. This compendium is assembled by noted molecular biologist and biochemist Nancy Smyth Templeton. Baylor College of Medicine and several other institutions have used Dr. Templeton’s non-viral therapeutics in clinical trials for the treatment of lung, breast, head and neck, and pancreatic cancers, as well as Hepatitis B and C. She continues to work at the forefront of research in gene and cell therapies. Her contributions, as well as those contained in this volume, are sure to advance the state of the art of these revolutionary life-saving technologies.

Advances In Pharmaceutical Cell Therapy: Principles Of Cell-based Biopharmaceuticals

Advances In Pharmaceutical Cell Therapy: Principles Of Cell-based Biopharmaceuticals
  • Author : Huss Ralf,Guenther Christine,Hauser Andrea Josefine
  • Publisher :Unknown
  • Release Date :2015-09-23
  • Total pages :564
  • ISBN : 9789814616805
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Summary : This textbook is a comprehensive overview of the development of cell-based biopharmaceuticals. Beginning with the underlying biology of stem cell and cell-based products, it traces the long and complex journey from preclinical concept to initiation of a pivotal clinical trial and the potential business model behind it.The book also takes into consideration the different regulatory landscapes and their continuous evolution in Europe, North America and other parts of the world. The authors describe a path to manufacture a clinical grade therapeutic that passes all necessary quality measures as a robust and marketable product including an outlook on next generation products and innovative strategies.This reference book is a must-have guide for any professional already active in biopharmaceuticals and anyone interested in getting involved in a scientific, medical or business capacity.

Adenoviral Vectors for Gene Therapy

Adenoviral Vectors for Gene Therapy
  • Author : David T. Curiel
  • Publisher :Unknown
  • Release Date :2016-03-10
  • Total pages :868
  • ISBN : 9780128005101
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Summary : Adenoviral Vectors for Gene Therapy, Second Edition provides detailed, comprehensive coverage of the gene delivery vehicles that are based on the adenovirus that is emerging as an important tool in gene therapy. These exciting new therapeutic agents have great potential for the treatment of disease, making gene therapy a fast-growing field for research. This book presents topics ranging from the basic biology of adenoviruses, through the construction and purification of adenoviral vectors, cutting-edge vectorology, and the use of adenoviral vectors in preclinical animal models, with final consideration of the regulatory issues surrounding human clinical gene therapy trials. This broad scope of information provides a solid overview of the field, allowing the reader to gain a complete understanding of the development and use of adenoviral vectors. Provides complete coverage of the basic biology of adenoviruses, as well as their construction, propagation, and purification of adenoviral vectors Introduces common strategies for the development of adenoviral vectors, along with cutting-edge methods for their improvement Demonstrates noninvasive imaging of adenovirus-mediated gene transfer Discusses utility of adenoviral vectors in animal disease models Considers Federal Drug Administration regulations for human clinical trials

Safety and Efficacy of Gene-Based Therapeutics for Inherited Disorders

Safety and Efficacy of Gene-Based Therapeutics for Inherited Disorders
  • Author : Nicola Brunetti-Pierri
  • Publisher :Unknown
  • Release Date :2017-05-22
  • Total pages :220
  • ISBN : 9783319534572
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Summary : In this book, leading international experts analyze state-of-the-art advances in gene transfer vectors for applications in inherited disorders and also examine the toxicity profiles of these methods. The authors discuss the strengths and weaknesses of available vectors in the clinical setting, and specifically focus on the challenges and possible solutions that researchers are testing in order to improve the safety of gene therapy for genetic diseases. This comprehensive and authoritative overview of vector development is a necessary text for researchers, toxicologists, pharmacologists, molecular biologists, physicians, and students in these fields.

Modern Tools for Genetic Engineering

Modern Tools for Genetic Engineering
  • Author : Michael Kormann
  • Publisher :Unknown
  • Release Date :2016-05-18
  • Total pages :226
  • ISBN : 9789535123927
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Summary : Site-specific endonucleases create double-strand breaks within the genome and can be targeted to literally any genetic mutation. Together with a repair template, a correction of the defective locus becomes possible. This book offers insight into the modern tools of genome editing, their hurdles and their huge potential. A new era of in vivo genetic engineering has begun.

Essentials of Stem Cell Biology

Essentials of Stem Cell Biology
  • Author : Robert Lanza,John Gearhart,Brigid Hogan,Douglas Melton,Roger Pedersen,E. Donnall Thomas,James A. Thomson,Ian Sir Wilmut
  • Publisher :Unknown
  • Release Date :2009-06-05
  • Total pages :680
  • ISBN : 0080884970
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Summary : First developed as an accessible abridgement of the successful Handbook of Stem Cells, Essentials of Stem Cell Biology serves the needs of the evolving population of scientists, researchers, practitioners and students that are embracing the latest advances in stem cells. Representing the combined effort of seven editors and more than 200 scholars and scientists whose pioneering work has defined our understanding of stem cells, this book combines the prerequisites for a general understanding of adult and embryonic stem cells with a presentation by the world's experts of the latest research information about specific organ systems. From basic biology/mechanisms, early development, ectoderm, mesoderm, endoderm, methods to application of stem cells to specific human diseases, regulation and ethics, and patient perspectives, no topic in the field of stem cells is left uncovered. Selected for inclusion in Doody's Core Titles 2013, an essential collection development tool for health sciences libraries Contributions by Nobel Laureates and leading international investigators Includes two entirely new chapters devoted exclusively to induced pluripotent stem (iPS) cells written by the scientists who made the breakthrough Edited by a world-renowned author and researcher to present a complete story of stem cells in research, in application, and as the subject of political debate Presented in full color with glossary, highlighted terms, and bibliographic entries replacing references

Human Genome Editing

Human Genome Editing
  • Author : National Academies of Sciences, Engineering, and Medicine,National Academy of Medicine,National Academy of Sciences,Committee on Human Gene Editing: Scientific, Medical, and Ethical Considerations
  • Publisher :Unknown
  • Release Date :2017-08-13
  • Total pages :328
  • ISBN : 9780309452885
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Summary : 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.

Cancer Gene Therapy

Cancer Gene Therapy
  • Author : David T. Curiel,Joanne T. Douglas
  • Publisher :Unknown
  • Release Date :2007-11-03
  • Total pages :490
  • ISBN : 9781592597857
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Summary : A complete introduction and guide to the latest developments in cancer gene therapy-from bench to bedside. The authors comprehensively review the anticancer genes and gene delivery methods currently available for cancer gene therapy, including the transfer of genetic material into the cancer cells, stimulation of the immune system to recognize and eliminate cancer cells, and the targeting of the nonmalignant stromal cells that support their growth. They also thoroughly examine the advantages and limitations of the different therapies and detail strategies to overcome obstacles to their clinical implementation. Topics of special interest include vector-targeting techniques, the lessons learned to date from clinical trials of cancer gene therapy, and the regulatory guidelines for future trials. Noninvasive techniques to monitor the extent of gene transfer and disease regression during the course of treatment are also discussed.

Cell and Gene Therapies

Cell and Gene Therapies
  • Author : Miguel-Angel Perales,Syed A. Abutalib,Catherine Bollard
  • Publisher :Unknown
  • Release Date :2018-11-27
  • Total pages :288
  • ISBN : 9783319543680
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Summary : In this book, experts in the field express their well-reasoned opinions on a range of complex, clinically relevant issues across the full spectrum of cell and gene therapies with the aim of providing trainee and practicing hematologists, including hematopoietic transplant physicians, with information that is relevant to clinical practice and ongoing research. Each chapter focuses on a particular topic, and the concise text is supported by numerous working tables, algorithms, and figures. Whenever appropriate, guidance is provided regarding the availability of potentially high-impact clinical trials. The rapid evolution of cell and gene therapies is giving rise to numerous controversies that need to be carefully addressed. In meeting this challenge, this book will appeal to all residents, fellows, and faculty members responsible for the care of hematopoietic cell transplant patients. It will also offer a robust, engaging tool to aid vital activities in the daily work of every hematology and oncology trainee.

Stem Cells in Regenerative Medicine

Stem Cells in Regenerative Medicine
  • Author : Alain A. Vertes,Nasib Qureshi,Arnold I. Caplan,Lee E. Babiss
  • Publisher :Unknown
  • Release Date :2015-09-14
  • Total pages :776
  • ISBN : 9781118846216
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Summary : This book is a unique guide to emerging stem cell technologies and the opportunities for their commercialisation. It provides in-depth analyses of the science, business, legal, and financing fundamentals of stem cell technologies, offering a holistic assessment of this emerging and dynamic segment of the field of regenerative medicine. • Reviews the very latest advances in the technology and business of stem cells used for therapy, research, and diagnostics • Identifies key challenges to the commercialisation of stem cell technology and avenues to overcome problems in the pipeline • Written by an expert team with extensive experience in the business, basic and applied science of stem cell research This comprehensive volume is essential reading for researchers in cell biology, biotechnology, regenerative medicine, and tissue engineering, including scientists and professionals, looking to enter commercial biotechnology fields.

Cardiac Cell and Gene Transfer

Cardiac Cell and Gene Transfer
  • Author : Joseph M. Metzger
  • Publisher :Unknown
  • Release Date :2003
  • Total pages :253
  • ISBN : 9781592593507
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Summary : A panel of leading scientific experts detail novel techniques and strategies for the cellular and genetic modification of heart function. The highly experienced authors provide step-by-step protocols for vector production and purification, for gene and cell delivery techniques, and for physiological assessment in vivo and in vitro. Timely, authoritative, and state-of-the-art, Cardiac Cell and Gene Transfer: Principles, Protocols, and Applications constitutes an invaluable guide to all the new cellular and genebased technologies needed by basic and clinical investigators working to illuminate today's unanswered questions about heart disease and ultimately to improve the heart performance in all their patients.

Pharmaceutical Biotechnology

Pharmaceutical Biotechnology
  • Author : Gary Walsh
  • Publisher :Unknown
  • Release Date :2013-04-25
  • Total pages :504
  • ISBN : 9781118685754
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Summary : Pharmaceutical Biotechnology offers students taking Pharmacy and related Medical and Pharmaceutical courses a comprehensive introduction to the fast-moving area of biopharmaceuticals. With a particular focus on the subject taken from a pharmaceutical perspective, initial chapters offer a broad introduction to protein science and recombinant DNA technology- key areas that underpin the whole subject. Subsequent chapters focus upon the development, production and analysis of these substances. Finally the book moves on to explore the science, biotechnology and medical applications of specific biotech products categories. These include not only protein-based substances but also nucleic acid and cell-based products. introduces essential principles underlining modern biotechnology- recombinant DNA technology and protein science an invaluable introduction to this fast-moving subject aimed specifically at pharmacy and medical students includes specific ‘product category chapters’ focusing on the pharmaceutical, medical and therapeutic properties of numerous biopharmaceutical products. entire chapter devoted to the principles of genetic engineering and how these drugs are developed. includes numerous relevant case studies to enhance student understanding no prior knowledge of protein structure is assumed

Gene Therapy

Gene Therapy
  • Author : Doaa Hashad
  • Publisher :Unknown
  • Release Date :2015-11-26
  • Total pages :218
  • ISBN : 9789535122210
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Summary : Gene therapy is becoming a promising technology for the management of many human diseases. Hereditary and acquired disorders can both be tackled using the technique of gene therapy. This book provides detailed, up-to-date topics addressing basic principles of gene therapy and discussing some of the challenges encountered by scientists in developing this relatively novel technology. The development of new and efficient gene transfer vectors is of utmost importance in the progress of the field of gene therapy. Both viral and non-viral vectors are extensively discussed. A detailed chapter elaborates the problem of host immune rejection of transplanted donor cells or engineered tissue that can be avoided using the encapsulation of transgenic cells, thus avoiding the use of drugs that achieve immunosuppression.

Gaucher Disease

Gaucher Disease
  • Author : Anthony H. Futerman,Ari Zimran
  • Publisher :Unknown
  • Release Date :2006-07-07
  • Total pages :544
  • ISBN : 9781420005509
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Summary : In September of 2007 Gaucher Disease received a commendation in the Haematology category of the 2007 British Medical Association Medical Book Competition! Although rare in the general population, Gaucher disease is the most prevalent of the lysosomal storage disorders, making research into this particular orphan disorder an invaluable prototype for the diagnosis, research, and treatment of others. Like many rare conditions, Gaucher disease has benefited from the explosion of medical research in the last decade, the amount of new information on this disease is staggering and the rate of new discoveries has left previous books on the subject unable to provide useful, up-to-date information. The most current, fully comprehensive reference to date, Gaucher Disease provides valuable information for academic and industry scientists, and clinicians. Outlining the latest research on the biochemical mechanisms and pathology of lysosomal storage disorders, this book covers diverse topics including animal models, crystallography, imaging and radionuclide evaluation. It not only addresses the developmental basis for current treatments like the now widely available enzyme replacement therapy, but also includes chapters introducing new therapies on the horizon. With contributions from world-renowned experts in substrate reduction therapy, pharmaceutical chaperone therapy, hematopoietic stem cell transplantation and gene therapy, as well as chapters on a second generation of enzyme replacement therapy, this book explores the full spectrum of possibilities offered by the most recent advances in medicine. Some of the most interesting aspects of the book include the discussions on patient management, those touching on the ethics of research, and the societal aspects of treating rare diseases with expensive therapy.

Muscle Gene Therapy

Muscle Gene Therapy
  • Author : Dongsheng Duan
  • Publisher :Unknown
  • Release Date :2009-11-26
  • Total pages :275
  • ISBN : 144191207X
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Summary : Muscle disease represents an important health threat to the general population. There is essentially no cure. Gene therapy holds great promise to correct the genetic defects and eventually achieve full recovery in these diseases. Significant progresses have been made in the field of muscle gene therapy over the last few years. The development of novel gene delivery vectors has substantially enhanced specificity and efficiency of muscle gene delivery. The new knowledge on the immune response to viral vectors has added new insight in overcoming the immune obstacles. Most importantly, the field has finally moved from small experimental animal models to human patients. This book will bring together the leaders in the field of muscle gene transfer to provide an updated overview on the progress of muscle gene therapy. It will also highlight important clinical applications of muscle gene therapy.