Exon Skipping And Inclusion Therapies
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Exon Skipping And Inclusion Therapies
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Author : Toshifumi Yokota
language : en
Publisher:
Release Date : 2018
Exon Skipping And Inclusion Therapies written by Toshifumi Yokota and has been published by this book supported file pdf, txt, epub, kindle and other format this book has been release on 2018 with Biomedicine categories.
Exon Skipping And Inclusion Therapies
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Author : Toshifumi Yokota
language : en
Publisher: Humana Press
Release Date : 2018-09-01
Exon Skipping And Inclusion Therapies written by Toshifumi Yokota and has been published by Humana Press this book supported file pdf, txt, epub, kindle and other format this book has been release on 2018-09-01 with Medical categories.
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.
Muscle Gene Therapy
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Author : Dongsheng Duan
language : en
Publisher: Springer
Release Date : 2014-09-09
Muscle Gene Therapy written by Dongsheng Duan and has been published by Springer this book supported file pdf, txt, epub, kindle and other format this book has been release on 2014-09-09 with Medical categories.
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.
Nonsense Mutation Correction In Human Diseases
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Author : Fabrice Lejeune
language : en
Publisher: Academic Press
Release Date : 2016-02-26
Nonsense Mutation Correction In Human Diseases written by Fabrice Lejeune and has been published by Academic Press this book supported file pdf, txt, epub, kindle and other format this book has been release on 2016-02-26 with Science categories.
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
Challenges And Opportunities Of Targeting Muscle For Therapy
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Author : Carlo Rinaldi
language : en
Publisher: Frontiers Media SA
Release Date : 2022-03-30
Challenges And Opportunities Of Targeting Muscle For Therapy written by Carlo Rinaldi and has been published by Frontiers Media SA this book supported file pdf, txt, epub, kindle and other format this book has been release on 2022-03-30 with Science categories.
Duchenne Muscular Dystrophy
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Author : Camilla Bernardini
language : en
Publisher: Humana Press
Release Date : 2017-10-25
Duchenne Muscular Dystrophy written by Camilla Bernardini and has been published by Humana Press this book supported file pdf, txt, epub, kindle and other format this book has been release on 2017-10-25 with Science categories.
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.
Rna Splicing And Backsplicing Disease And Therapy
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Author : Rosanna Asselta
language : en
Publisher: Frontiers Media SA
Release Date : 2020-12-24
Rna Splicing And Backsplicing Disease And Therapy written by Rosanna Asselta and has been published by Frontiers Media SA this book supported file pdf, txt, epub, kindle and other format this book has been release on 2020-12-24 with Science categories.
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.
Genome Editing In Neurosciences
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Author : Rudolf Jaenisch
language : en
Publisher:
Release Date : 2020-10-08
Genome Editing In Neurosciences written by Rudolf Jaenisch and has been published by this book supported file pdf, txt, epub, kindle and other format this book has been release on 2020-10-08 with Medical categories.
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.
Muscular Dystrophy Therapeutics
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Author : Rika Maruyama
language : en
Publisher: Springer Nature
Release Date : 2022-11-18
Muscular Dystrophy Therapeutics written by Rika Maruyama and has been published by Springer Nature this book supported file pdf, txt, epub, kindle and other format this book has been release on 2022-11-18 with Medical categories.
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.
Neuromuscular Disorders
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Author : Ashraf Zaher
language : en
Publisher: IntechOpen
Release Date : 2012-08-01
Neuromuscular Disorders written by Ashraf Zaher and has been published by IntechOpen this book supported file pdf, txt, epub, kindle and other format this book has been release on 2012-08-01 with Medical categories.
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.