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Author : Toshifumi Yokota
Publisher :
ISBN 13 : 9781493986514
Total Pages : 569 pages
Book Rating : 4.9/5 (865 download)
Download or read book Exon Skipping and Inclusion Therapies written by Toshifumi Yokota and published by . This book was released on 2018 with total page 569 pages. Available in PDF, EPUB and Kindle. Book excerpt:
Author : Toshifumi Yokota
Publisher : Humana Press
ISBN 13 : 9781493986507
Total Pages : 569 pages
Book Rating : 4.9/5 (865 download)
Download or read book Exon Skipping and Inclusion Therapies written by Toshifumi Yokota and published by Humana Press. This book was released on 2018-09-01 with total page 569 pages. Available in PDF, EPUB and Kindle. Book excerpt: This book presents a comprehensive collection of detailed state-of-the-art exon skipping and splices modulation protocols. Chapters detail 14 genetic diseases, AON-mediated therapies, and CRISPR/Cas9-mediated gene editing therapies. Written in the highly successful Methods in Molecular Biology series format, chapters include introductions to their respective topics, lists of the necessary materials and reagents, step-by-step, readily reproducible laboratory protocols, and tips on troubleshooting and avoiding known pitfalls. Authoritative and cutting-edge, Exon Skipping and Inclusion Therapies: Methods and Protocols aims to help researchers initiate the development of next-generation therapies.
Author : Ashraf Zaher
Publisher : IntechOpen
ISBN 13 : 9789535106968
Total Pages : 284 pages
Book Rating : 4.1/5 (69 download)
Download or read book Neuromuscular Disorders written by Ashraf Zaher and published by IntechOpen. This book was released on 2012-08-01 with total page 284 pages. Available in PDF, EPUB and Kindle. Book excerpt: Neuromuscular Disorders is an interesting compendium of diverse and fairly inclusive topics in disorders of nerve and muscle, with a spectrum of generally well written and pertinent chapters referencing specific categories of neuromuscular diseases. The expected emphasis on understanding the implications, diagnosis and treatment of the specific muscle and nerve diseases is well done. It contains a basic introductory chapter which is very well-done, and a large range of topics, well-summarized, making it a good reference manual as well as an aid to diagnosis and treatment contained in a relatively small volume. Its accessibility on the internet may make it especially appealing to the younger of us, making it simple to access without necessitating the book's presence in the office.
Author : Fabrice Lejeune
Publisher : Academic Press
ISBN 13 : 0128044691
Total Pages : 192 pages
Book Rating : 4.1/5 (28 download)
Download or read book Nonsense Mutation Correction in Human Diseases written by Fabrice Lejeune and published by Academic Press. This book was released on 2016-02-26 with total page 192 pages. Available in PDF, EPUB and Kindle. Book excerpt: Nonsense Mutation Correction in Human Diseases: An Approach for Targeted Medicine provides an introduction on genetic diseases, discusses the prevalence of nonsense mutations, the consequences of a nonsense mutation for the expression of the mutant gene, and the presentation of the nonsense-mediated mRNA decay (NMD). It presents the mechanism of action and rationale associated with each strategy to correct nonsense mutations with the results of clinical trials to further support this basis. In addition, the book shows how it may be possible to combine several of these strategies to ultimately improve the efficiency of correction, also suggesting the future goals and objectives to improve treatment modalities in this evolving sphere of personalized medicine. Features basic biological and clinical constructs that inform the application of genomic data to clinical decision-making Includes theories and methods that can be used to link bio-molecular and clinical phenotypes so as to enable integrative hypothesis discovery, testing, and downstream evidence-based practice Provides design patterns and use cases that contextualize the clinical decision-making and evidence-based practice relative to real world requirements and stakeholders
Author : Ashraf Zaher
Publisher : IntechOpen
ISBN 13 : 9789535106968
Total Pages : 284 pages
Book Rating : 4.1/5 (69 download)
Download or read book Neuromuscular Disorders written by Ashraf Zaher and published by IntechOpen. This book was released on 2012-08-01 with total page 284 pages. Available in PDF, EPUB and Kindle. Book excerpt: Neuromuscular Disorders is an interesting compendium of diverse and fairly inclusive topics in disorders of nerve and muscle, with a spectrum of generally well written and pertinent chapters referencing specific categories of neuromuscular diseases. The expected emphasis on understanding the implications, diagnosis and treatment of the specific muscle and nerve diseases is well done. It contains a basic introductory chapter which is very well-done, and a large range of topics, well-summarized, making it a good reference manual as well as an aid to diagnosis and treatment contained in a relatively small volume. Its accessibility on the internet may make it especially appealing to the younger of us, making it simple to access without necessitating the book's presence in the office.
Author : Dongsheng Duan
Publisher : Springer Science & Business Media
ISBN 13 : 144191207X
Total Pages : 281 pages
Book Rating : 4.4/5 (419 download)
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 281 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.
Author : Camilla Bernardini
Publisher : Humana Press
ISBN 13 : 9781493973736
Total Pages : 287 pages
Book Rating : 4.9/5 (737 download)
Download or read book Duchenne Muscular Dystrophy written by Camilla Bernardini and published by Humana Press. This book was released on 2017-10-25 with total page 287 pages. Available in PDF, EPUB and Kindle. Book excerpt: This volume explores experimental approaches used to study Duchenne muscular dystrophy (DMD), an X-linked degenerative skeletal muscle disease caused by mutations in the dystrophin gene. Including the latest progress and scientific achievements, the book covers recent discoveries achieved through in vivo gene editing which have proven to be promising in restoring dystrophin expression, at least in ameliorating skeletal muscle symptoms, and the contents focus on “Omics” techniques in gene expression, protein expression, miRNAs, and long non-coding RNA analysis, as well as experimental studies of the structural/functional changes affecting the skeletal and cardiac muscles and ongoing preclinical studies and clinical trials. Written in the highly successful Methods in Molecular Biology series format, chapters include introductions to their respective topics, lists of the necessary materials and reagents, step-by-step, readily reproducible laboratory protocols, and tips on troubleshooting and avoiding known pitfalls. Authoritative and practical, Duchenne Muscular Dystrophy: Methods and Protocols serves as a guide for researchers exploring the complicated nature of dystrophin in the hope of helping the victims of this disorder.
Author : Rosanna Asselta
Publisher : Frontiers Media SA
ISBN 13 : 2889662357
Total Pages : 197 pages
Book Rating : 4.8/5 (896 download)
Download or read book RNA Splicing and Backsplicing: Disease and Therapy written by Rosanna Asselta and published by Frontiers Media SA. This book was released on 2020-12-24 with total page 197 pages. Available in PDF, EPUB and Kindle. Book excerpt: This eBook is a collection of articles from a Frontiers Research Topic. Frontiers Research Topics are very popular trademarks of the Frontiers Journals Series: they are collections of at least ten articles, all centered on a particular subject. With their unique mix of varied contributions from Original Research to Review Articles, Frontiers Research Topics unify the most influential researchers, the latest key findings and historical advances in a hot research area! Find out more on how to host your own Frontiers Research Topic or contribute to one as an author by contacting the Frontiers Editorial Office: frontiersin.org/about/contact.
Author : David Elliott
Publisher : Oxford University Press
ISBN 13 : 019107103X
Total Pages : 445 pages
Book Rating : 4.1/5 (91 download)
Download or read book Molecular Biology of RNA written by David Elliott and published by Oxford University Press. This book was released on 2017-01-31 with total page 445 pages. Available in PDF, EPUB and Kindle. Book excerpt: RNA plays a central, and until recently, somewhat underestimated role in the genetics underlying all forms of life on earth. This versatile molecule not only plays a crucial part in the synthesis of proteins from a DNA template, but is also intrinsically involved in the regulation of gene expression, and can even act as a catalyst in the form of a ribozyme. This latter property has led to the hypothesis that RNA - rather than DNA - could have played an essential part in the origin of life itself. This landmark text provides a systematic overview of the exciting and rapidly moving field of RNA biology. Key pioneering experiments, which provided the underlying evidence for what we now know, are described throughout, while the relevance of the subject to human disease is highlighted via frequent boxes. For the second edition of Molecular Biology of RNA, more introductory material has been incorporated at the beginning of the text, to aid students studying the subject for the first time. Throughout the text, new material has been included - particularly in relation to RNA binding domains, non-coding RNAs, and the connection between RNA biology and epigenetics. Finally, a new closing chapter discusses how exciting new technologies are being used to explore current topical areas of research.
Author : Basil T. Darras
Publisher : Elsevier
ISBN 13 : 0124171273
Total Pages : 1156 pages
Book Rating : 4.1/5 (241 download)
Download or read book Neuromuscular Disorders of Infancy, Childhood, and Adolescence written by Basil T. Darras and published by Elsevier. This book was released on 2014-12-03 with total page 1156 pages. Available in PDF, EPUB and Kindle. Book excerpt: Neuromuscular disorders are diagnosed across the lifespan and create many challenges especially with infants, children and adolescents. This new edition of the definitive reference, edited by the established world renowned authorities on the science, diagnosis and treatment of neuromuscular disorders in childhood is a timely and needed resource for all clinicians and researchers studying neuromuscular disorders, especially in childhood. The Second Edition is completely revised to remain current with advances in the field and to insure this remains the standard reference for clinical neurologists and clinical research neurologists. The Second Edition retains comprehensive coverage while shortening the total chapter count to be an even more manageable and effective reference. Carefully revised new edition of the classic reference on neuromuscular disorders in infancy, childhood and adolescence. Definitive coverage of the basic science of neuromuscular disease and the latest diagnosis and treatment best practices. Includes coverage of clinical phenomenology, electrophysiology, histopathology, molecular genetics and protein chemistry
Author : Roland W. Herzog
Publisher : World Scientific
ISBN 13 : 9814280917
Total Pages : 415 pages
Book Rating : 4.8/5 (142 download)
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
Author : Philippe Jeanteur
Publisher : Springer Science & Business Media
ISBN 13 : 3540344497
Total Pages : 265 pages
Book Rating : 4.5/5 (43 download)
Download or read book Alternative Splicing and Disease written by Philippe Jeanteur and published by Springer Science & Business Media. This book was released on 2006-10-04 with total page 265 pages. Available in PDF, EPUB and Kindle. Book excerpt: Splicing of primary RNA transcript is a quasi-systematic step of gene expression in higher organisms. This is the first book to highlight the medical implications, i.e. diseases, caused by alternative splicing. Alternative splicing not only vastly increases protein diversity but also offers numerous opportunities for aberrant splicing events with pathological consequences. The book also outlines possible targets for therapy.
Author : Roger N. Rosenberg
Publisher : Elsevier
ISBN 13 : 0124105491
Total Pages : 1465 pages
Book Rating : 4.1/5 (241 download)
Download or read book Rosenberg's Molecular and Genetic Basis of Neurological and Psychiatric Disease written by Roger N. Rosenberg and published by Elsevier. This book was released on 2014-10-28 with total page 1465 pages. Available in PDF, EPUB and Kindle. Book excerpt: Rosenberg’s Molecular and Genetic Basis of Neurologic and Psychiatric Disease, Fifth Edition provides a comprehensive introduction and reference to the foundations and key practical aspects relevant to the majority of neurologic and psychiatric disease. A favorite of over three generations of students, clinicians and scholars, this new edition retains and expands the informative, concise and critical tone of the first edition. This is an essential reference for general medical practitioners, neurologists, psychiatrists, geneticists, and related professionals, and for the neuroscience and neurology research community. The content covers all aspects essential to the practice of neurogenetics to inform clinical diagnosis, treatment and genetic counseling. Every chapter has been thoroughly revised or newly commissioned to reflect the latest scientific and medical advances by an international team of leading scientists and clinicians. The contents have been expanded to include disorders for which a genetic basis has been recently identified, together with abundant original illustrations that convey and clarify the key points of the text in an attractive, didactic format. Previous editions have established this book as the leading tutorial reference on neurogenetics. Researchers will find great value in the coverage of genomics, animal models and diagnostic methods along with a better understanding of the clinical implications. Clinicians will rely on the coverage of the basic science of neurogenetics and the methods for evaluating patients with biochemical abnormalities or gene mutations, including links to genetic testing for specific diseases. Comprehensive coverage of the neurogenetic foundation of neurological and psychiatric disease Detailed introduction to both clinical and basic research implications of molecular and genetic understanding of the brain Detailed coverage of genomics, animal models and diagnostic methods with new coverage of evaluating patients with biochemical abnormalities or gene mutations
Author :
Publisher : Academic Press
ISBN 13 : 0323853021
Total Pages : 560 pages
Book Rating : 4.3/5 (238 download)
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
Author : Rudolf Jaenisch
Publisher :
ISBN 13 : 9781013268564
Total Pages : 128 pages
Book Rating : 4.2/5 (685 download)
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.
Author : Carlo Rinaldi
Publisher : Frontiers Media SA
ISBN 13 : 2889747840
Total Pages : 128 pages
Book Rating : 4.8/5 (897 download)
Download or read book Challenges and Opportunities of Targeting Muscle for Therapy written by Carlo Rinaldi and published by Frontiers Media SA. This book was released on 2022-03-30 with total page 128 pages. Available in PDF, EPUB and Kindle. Book excerpt:
Author : Rika Maruyama
Publisher : Springer Nature
ISBN 13 : 1071627724
Total Pages : 568 pages
Book Rating : 4.0/5 (716 download)
Download or read book Muscular Dystrophy Therapeutics written by Rika Maruyama and published by Springer Nature. This book was released on 2022-11-18 with total page 568 pages. Available in PDF, EPUB and Kindle. Book excerpt: This detailed book presents a comprehensive collection of state-of-the-art protocols on muscular dystrophy therapeutics, covering therapeutics using antisense oligonucleotides, gene replacement, genome editing, small molecules, stem cells, and antibodies. Written by leaders in the field, the volume explores techniques that are currently in use and are starting an exciting therapeutic revolution in muscular dystrophy. As a part of the highly successful Methods in Molecular Biology series, chapters include introductions to their respective topics, lists of the necessary materials and reagents, step-by-step and readily reproducible laboratory protocols, as well as tips on troubleshooting and avoiding known pitfalls. Authoritative and practical, Muscular Dystrophy Therapeutics: Methods and Protocols serves as an ideal resource to inspire readers and provide tips, strategies, and advice to develop new therapeutic technologies for this group of diseases.
