Muscle Gene Therapy

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Muscle Gene Therapy

Author : Dongsheng Duan
Publisher : Springer Science & Business Media
ISBN 13 : 144191207X
Total Pages : 275 pages
Book Rating : 4.4/5 (419 download)

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Book Synopsis Muscle Gene Therapy by : Dongsheng Duan

Download or read book Muscle Gene Therapy written by Dongsheng Duan and published by Springer Science & Business Media. This book was released on 2009-11-26 with total page 275 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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.

Genome Editing in Neurosciences

Author : Rudolf Jaenisch
Publisher : Springer
ISBN 13 : 9783319868011
Total Pages : 0 pages
Book Rating : 4.8/5 (68 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 Springer. This book was released on 2018-08-11 with total page 0 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.

Muscle Gene Therapy

Author : Dongsheng Duan
Publisher : Humana Press
ISBN 13 : 9781493961825
Total Pages : 382 pages
Book Rating : 4.9/5 (618 download)

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Book Synopsis Muscle Gene Therapy by : Dongsheng Duan

Download or read book Muscle Gene Therapy written by Dongsheng Duan and published by Humana Press. This book was released on 2016-08-23 with total page 382 pages. Available in PDF, EPUB and Kindle. Book excerpt: Gene therapy offers many conceptual advantages to treat muscle diseases, especially various forms of muscular dystrophies; however, it faces a number of unique challenges, including the need to deliver a therapeutic vector to all muscles throughout the body. In Muscle Gene Therapy: Methods and Protocols, expert researchers in the field present a collection of techniques aimed at bridging the translational gap in muscle gene therapy between the prevalent rodent models and vitally important larger animal models. Divided into three sections, this volume examines basic protocols for optimizing the muscle gene expression cassette and for evaluating the therapeutic outcomes, new developments in muscle gene therapy technology such as adeno-associated viral vector (AAV), oligonucleotide-mediated exon-skipping, and novel RNA-based strategies, and step-by-step guidance on muscle gene delivery in swine, ovine, canine, and non-human primates. Written in the highly successful Methods in Molecular BiologyTM series format, chapters include introductions to their respective topics, lists of the necessary materials and reagents, detailed, readily reproducible laboratory protocols, and tips on troubleshooting and avoiding known pitfalls. Authoritative and cutting-edge, Muscle Gene Therapy: Methods and Protocols serves as an invaluable resource for graduate students, post-doctoral fellows, and principle investigators pursuing the crucial advancement of muscle disease gene therapy in the hope of someday curing these debilitating disorders.

The Muscular Dystrophies

Author : Alan E. H. Emery
Publisher :
ISBN 13 :
Total Pages : 338 pages
Book Rating : 4.3/5 (91 download)

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Book Synopsis The Muscular Dystrophies by : Alan E. H. Emery

Download or read book The Muscular Dystrophies written by Alan E. H. Emery and published by . This book was released on 2001 with total page 338 pages. Available in PDF, EPUB and Kindle. Book excerpt: The muscular dystrophies are an important group of inherited disorders. They are characterized by muscle wasting and weakness, but vary considerably in their clinical manifestations and severity. This text reviews our understanding of the most important of these disorders. In many instances, the genes and protein products responsible for the dystrophies have been identified and it is now possible to establish a precise diagnosis, detect preclinical cases, identify carriers and offer prenatal dianostic testing. The book goes on to describe the opportunities for management of the symptoms through respiratory care, physiotherapy and surgical correction of contracture, and examines the potential, in the future, for effective treatment utilizing the new techniques of gene and cell therapy. Professor Emery has invited chapters from the leading international experts in the field providing a unique insight into the current situation and the hopes for the future.

Duchenne Muscular Dystrophy

Author : Jeffrey S. Chamberlain
Publisher : CRC Press
ISBN 13 : 0849374456
Total Pages : 496 pages
Book Rating : 4.8/5 (493 download)

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Book Synopsis Duchenne Muscular Dystrophy by : Jeffrey S. Chamberlain

Download or read book Duchenne Muscular Dystrophy written by Jeffrey S. Chamberlain and published by CRC Press. This book was released on 2006-02-27 with total page 496 pages. Available in PDF, EPUB and Kindle. Book excerpt: Duchenne Muscular Dystrophy (DMD) is one of the most prevalent genetic disorders of childhood and currently stands as an incurable condition. This authoritative guide provides a clear overview of the latest current and experimental approaches to the treatment of DMD and examines the clinical, genetic, and pathophysiological aspects of the disease i

Spinal Muscular Atrophy

Author : Charlotte J Sumner
Publisher : Academic Press
ISBN 13 : 0128036869
Total Pages : 506 pages
Book Rating : 4.1/5 (28 download)

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Book Synopsis Spinal Muscular Atrophy by : Charlotte J Sumner

Download or read book Spinal Muscular Atrophy written by Charlotte J Sumner and published by Academic Press. This book was released on 2016-10-24 with total page 506 pages. Available in PDF, EPUB and Kindle. Book excerpt: Spinal Muscular Atrophy: Disease Mechanisms and Therapy provides the latest information on a condition that is characterized by motoneuron loss and muscle atrophy, and is the leading genetic cause of infant mortality. Since the identification of the gene responsible for SMA in 1995, there have been important advances in the basic understanding of disease mechanisms, and in therapeutic development. This book provides a comprehensive accounting of recent advances in basic and clinical research that covers SMA clinical features and standards of care, multifaceted aspects of SMN protein functions and SMA disease pathology, various animal models, and biomarkers, as well as current therapeutic development. This title is ideal for graduate students/postdocs and principal investigators who are already in the SMA field and need to keep updated on recent findings and approaches, and for those who are new to, or would like to join, the field. Likewise, users will find an excellent source of reading for biotech/pharma scientists, clinical researchers, and practitioners, regulators, and patients and their advocacy organizations. Furthermore, this book is a handy reference for researchers and clinicians who may want to apply the research strategies and therapeutic approaches in SMA to other rare diseases. Provides comprehensive, up-to-date reviews by leading investigators on diverse topics of SMA, including clinical features and patient care, SMN genetics and protein functions, animal models, disease pathology and mechanisms, biomarkers, current therapeutic development, and the role of non-profit organizations in therapeutic development Written to bridge multiple disciplines and promote better communications among basic scientists, clinical researchers, and health care providers on the latest developments in SMA Includes outstanding questions and perspectives for future investigations and key references for additional detailed study

Muscular Dystrophy

Author : Paula Johanson
Publisher : The Rosen Publishing Group, Inc
ISBN 13 : 1435848241
Total Pages : 66 pages
Book Rating : 4.4/5 (358 download)

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Book Synopsis Muscular Dystrophy by : Paula Johanson

Download or read book Muscular Dystrophy written by Paula Johanson and published by The Rosen Publishing Group, Inc. This book was released on 2008-08-15 with total page 66 pages. Available in PDF, EPUB and Kindle. Book excerpt: Muscular dystrophy (MD) is a genetic disorder that gradually weakens a persons muscles. It is caused by missing or incorrect genetic information in the bodys cells. The persons body is unable to make the proteins needed to build and maintain healthy muscles. Although there is no cure for muscular dystrophy, researchers are learning about how to prevent and treat the condition. Doctors have learned a great deal about how to improve muscle and joint function and how to slow the deterioration of muscles. Their goal is to help children, teenagers, and adults with muscular dystrophy live active and independent lives for as long as possible. This book delves into the history of the disorder, the genetic basis of MD, the main types of MD, and living with MD and suggests some coping strategies. It also explores diagnostic tests for MD, genetic research, and the promising effects of gene therapy.

Duchenne Muscular Dystrophy

Author : Alan E. H. Emery
Publisher : OUP Oxford
ISBN 13 : 0191503665
Total Pages : 320 pages
Book Rating : 4.1/5 (915 download)

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Book Synopsis Duchenne Muscular Dystrophy by : Alan E. H. Emery

Download or read book Duchenne Muscular Dystrophy written by Alan E. H. Emery and published by OUP Oxford. This book was released on 2015-02-19 with total page 320 pages. Available in PDF, EPUB and Kindle. Book excerpt: Duchenne Muscular Dystrophy, an inherited and progressive muscle wasting disease, is one of the most common single gene disorders found in the developed world. In this fourth edition of the classic monograph on the topic, Alan Emery and Francesco Muntoni are joined by Rosaline Quinlivan, Consultant in Neuromuscular Disorders, to provide a thorough update on all aspects of the disorder. Recent understanding of the nature of the genetic defect responsible for Duchenne Muscular Dystrophy and isolation of the protein dystrophin has led to the development of new theories for the disease's pathogenesis. This new edition incorporates these advances from the field of molecular biology, and describes the resultant opportunities for screening, prenatal diagnosis, genetic counselling and from recent pioneering work with anti-sense oligonucleotides, the possibility of effective RNA therapy. Although there is still no cure for the disorder, there have been significant developments concerning the gene basis, publication of standards of care guidelines, and improvements in management leading to significantly longer survival, particularly with cardio-pulmonary care. The authors also investigate other forms of pharmacological, cellular and gene therapies. Duchenne Muscular Dystrophy will be essential reading not only for scientists and clinicians, but will also appeal to therapists and other professionals involved in the care of patients with muscular dystrophy.

Muscular Dystrophy

Author : National Institute of Neurological Diseases and Stroke
Publisher :
ISBN 13 :
Total Pages : 26 pages
Book Rating : 4.:/5 (319 download)

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Book Synopsis Muscular Dystrophy by : National Institute of Neurological Diseases and Stroke

Download or read book Muscular Dystrophy written by National Institute of Neurological Diseases and Stroke and published by . This book was released on 1971 with total page 26 pages. Available in PDF, EPUB and Kindle. Book excerpt:

Disorders of Voluntary Muscle

Author : George Karpati
Publisher : Cambridge University Press
ISBN 13 : 9780521650625
Total Pages : 800 pages
Book Rating : 4.6/5 (56 download)

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Book Synopsis Disorders of Voluntary Muscle by : George Karpati

Download or read book Disorders of Voluntary Muscle written by George Karpati and published by Cambridge University Press. This book was released on 2001-07-12 with total page 800 pages. Available in PDF, EPUB and Kindle. Book excerpt: Rewritten and redesigned, this remains the one essential text on the diseases of skeletal muscle.

Advancing Gene-Targeted Therapies for Central Nervous System Disorders

Author : National Academies of Sciences, Engineering, and Medicine
Publisher : National Academies Press
ISBN 13 : 0309495849
Total Pages : 93 pages
Book Rating : 4.3/5 (94 download)

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Book Synopsis Advancing Gene-Targeted Therapies for Central Nervous System Disorders by : National Academies of Sciences, Engineering, and Medicine

Download or read book Advancing Gene-Targeted Therapies for Central Nervous System Disorders written by National Academies of Sciences, Engineering, and Medicine and published by National Academies Press. This book was released on 2019-12-01 with total page 93 pages. Available in PDF, EPUB and Kindle. Book excerpt: On April 23 and 24, 2019 the Forum on Neuroscience and Nervous System Disorders convened a workshop titled "Advancing Gene-Targeted Therapies for Central Nervous System Disorders" in Washington, DC. This public workshop brought together experts and key stakeholders from academia, government, industry, philanthropic foundations, and disease/patient-focused nonprofit organizations to explore approaches for advancing the development of gene-targeted therapies for central nervous system (CNS) disorders, and implications of developing these therapies. Participants explored lessons learned from both successful and unsuccessful clinical development programs; new knowledge about the genetic underpinnings of brain disorders; the current status and future potential of gene-targeted therapies for CNS disorders; challenges and potential solutions for translating preclinical findings to approved therapies; and patient and caregiver perspectives. They also discussed what will be needed to develop these therapies for common disorders such as Alzheimer's and Parkinson's disease, as well as neuropsychiatric and neurodevelopmental disorders such as schizophrenia and autism. The workshop included approaches that target both DNA and RNA, as well as gene products using viral vectors, antisense oligonucleotides, and RNA interference. This publication summarizes the presentations and discussion of the workshop.

Muscular Dystrophy

Author : Gisela Gaina
Publisher : BoD – Books on Demand
ISBN 13 : 1839684747
Total Pages : 117 pages
Book Rating : 4.8/5 (396 download)

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Book Synopsis Muscular Dystrophy by : Gisela Gaina

Download or read book Muscular Dystrophy written by Gisela Gaina and published by BoD – Books on Demand. This book was released on 2020-12-23 with total page 117 pages. Available in PDF, EPUB and Kindle. Book excerpt: Muscular Dystrophy - Research Update and Therapeutic Strategies is for students, researchers, and clinicians interested in muscular dystrophies who want to improve their knowledge of these complex genetic diseases. The book includes information about the genetics of various types of muscular dystrophies as well as explores new and current therapeutic strategies that aim to ameliorate symptoms and improve patients’ quality of life and life expectancy. In addition, this book reviews information on current clinical trials for muscular dystrophies and presents a framework for what to consider during the design of these trials.

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

Gene Therapy

Author : David Cooper
Publisher : Garland Science
ISBN 13 : 1000144682
Total Pages : 431 pages
Book Rating : 4.0/5 (1 download)

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Book Synopsis Gene Therapy by : David Cooper

Download or read book Gene Therapy written by David Cooper and published by Garland Science. This book was released on 2020-09-10 with total page 431 pages. Available in PDF, EPUB and Kindle. Book excerpt: Gene Therapy describes the delivery systems now available to target a given tissue with specific gene or oligonucleotide sequences, and explores the utility of animal modules as test systems. In the context of selected disease states, it summarises in vitro and in vivo studies and clinical trials performed to date.

Stem Cell Biology and Gene Therapy

Author : Peter J. Quesenberry
Publisher : John Wiley & Sons
ISBN 13 : 9780471146568
Total Pages : 586 pages
Book Rating : 4.1/5 (465 download)

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Book Synopsis Stem Cell Biology and Gene Therapy by : Peter J. Quesenberry

Download or read book Stem Cell Biology and Gene Therapy written by Peter J. Quesenberry and published by John Wiley & Sons. This book was released on 1998-09-10 with total page 586 pages. Available in PDF, EPUB and Kindle. Book excerpt: STEM CELL BIOLOGY AND GENE THERAPY Edited by Peter J. Quesenberry, Gary S. Stein, Bernard Forget, and Sherman Weissman Advances in molecular genetics and recombinant DNA technology have ushered in a new era in medical therapeutic research. New insights into the molecular basis of human disease and the role played by biological regulatory mechanisms have precipitated tremendous drug development efforts backed by intensive research into human gene therapy worldwide. Stem Cell Biology and Gene Therapy is the first book to thoroughly cover major advances in the field and their applications to novel molecular therapies. This self-contained volume integrates biological and clinical components of stem cell biology, examines some of the most difficult aspects of gene therapy, and provides a systematic review of advanced gene modification techniques. Twenty essays by leading researchers address some of the most compelling topics in contemporary medical research, including: * Fundamental regulatory mechanisms that operate in stem cells * Stem cells from a therapeutic perspective, including preparations of stem cells and their therapeutic potential as vehicles for gene therapy * Delivery systems for therapeutic genes, including an overview of the most promising vectors * Clinical applications for gene therapy, covering a broad range of diseases such as hemophilia, cancers, neurological disease, and more Complete with illustrations and real-world examples of a variety of disorders, Stem Cell Biology and Gene Therapy is essential for researchers in gene therapy and members of the biotechnology industry who are developing human molecular therapies for commercial use. It is also an important reference for molecular biologists, cell biologists, immunologists, molecular geneticists, hematologists, cancer researchers, biochemists, and anyone working in internal medicine.

Myoblast Transfer Therapy

Author : Robert C. Griggs
Publisher : Springer Science & Business Media
ISBN 13 : 1468458655
Total Pages : 305 pages
Book Rating : 4.4/5 (684 download)

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Book Synopsis Myoblast Transfer Therapy by : Robert C. Griggs

Download or read book Myoblast Transfer Therapy written by Robert C. Griggs and published by Springer Science & Business Media. This book was released on 2012-12-06 with total page 305 pages. Available in PDF, EPUB and Kindle. Book excerpt: I am pleased to introduce this volume on Myoblast Transfer Therapy on behalf of the Muscular Dystrophy Association and all of its Advisory Committees. The international conference which led to this volume brought together leading basic scientists and clinical investigators for the purpose of coordinating the development of this new field in the fight against muscular dystrophy. The Muscular Dystrophy Association is the nation's most rapidly growing voluntary health agency in terms of its programs of patient care, research, and professional and public education. Success is attributable to its National Chairman, Jerry Lewis, to its effective corporate membership, and to the many physicians and scientists who give their time freely to advise on policies, to review grant applications, and to participate in meetings such as this. I should like to acknowledge a large number of other individuals to whom we are indebted: the broad segment of the American public which continually and generously supports our spectrum of services. The Muscular Dystrophy Association, next year, should raise in excess of $115,000,000. These contributions are derived from more than 10 million American families. These families are not only pledging their money but expressing their hopes that we will find answers to the tragic problem of neuromuscular disease. We are confident that the fruits of this meeting will move the frontier of research forward toward that goal.

Gene Therapy in Reconstructive and Regenerative Surgery

Author : Giorgio Giatsidis
Publisher : Springer
ISBN 13 : 9783030076894
Total Pages : 0 pages
Book Rating : 4.0/5 (768 download)

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Book Synopsis Gene Therapy in Reconstructive and Regenerative Surgery by : Giorgio Giatsidis

Download or read book Gene Therapy in Reconstructive and Regenerative Surgery written by Giorgio Giatsidis and published by Springer. This book was released on 2018-12-29 with total page 0 pages. Available in PDF, EPUB and Kindle. Book excerpt: This book offers an updated overview of the most recent research advances in the field, a comparison of established techniques and methods, a discussion on current experimental and translational challenges, and a commentary on potential opportunities and future directions. Dedicated chapters address and review the preclinical and clinical state-of-the-art of gene therapies for the reconstructive and regenerative surgery of skin and wounds, pathological scars, cartilage, tendons, skeletal muscles, and bio-engineered flaps. A brief guide to developing gene therapy clinical trials in the context of reconstructive and regenerative surgery is also provided. Biomedical and technological innovations are transforming our capacity to use gene therapies to safely and effectively repair, reconstruct, and regenerate tissues that are deficient or have been damaged by trauma and diseases. The targeted and controlled modulation of gene expression in tissues represents a game-changing, next-generation therapeutic tool for the modern reconstructive surgeon, expanding the horizon of regenerative surgery and tissue engineering. Through gene therapies, surgeons can direct (stem) cell differentiation and cell function, modulate the release of growth/transcriptional factors, affect the biological properties of regenerative scaffolds, control tissue inflammation, or induce immune-suppression in composite tissue allotransplants and xenotransplants. Written by renowned reconstructive surgeons and leading experts in each of these fields - from top academic institutions around the globe, the book provides an initial practical guide for veteran and newcomer surgeons alike, as well as for researchers interested in exploring the latest gene-based therapeutic strategies for reconstructive and regenerative surgery.