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Muscle Gene Therapy

Overview of attention for book
Cover of 'Muscle Gene Therapy'

Table of Contents

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    Book Overview
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    Chapter 1 An Overview of Muscle Biology and Physiology for Muscle Gene Therapy
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    Chapter 2 Molecular Basis of Muscle Disease
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    Chapter 3 Animal Models for Muscle Disease and Muscle Gene Therapy
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    Chapter 4 Muscle Stem Cell Biology and Implications in Gene Therapy
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    Chapter 5 Pluripotent Stem Cells for Gene Therapy of Hereditary Muscle Disorders
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    Chapter 6 MicroRNAs (miRs) in Muscle Gene Therapy
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    Chapter 7 Immune System Regulation of Muscle Injury and Disease
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    Chapter 8 Design of Muscle Gene Therapy Expression Cassette
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    Chapter 9 Non-viral Vector for Muscle-Mediated Gene Therapy
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    Chapter 10 Viral Vectors for Muscle Gene Therapy
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    Chapter 11 Development of Next-Generation Muscle Gene Therapy AAV Vectors
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    Chapter 12 Histological and Biochemical Evaluation of Muscle Gene Therapy
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    Chapter 13 Optical Polarization Tractography Imaging of Structural Changes in the Skeletal and Cardiac Muscles of the mdx4cv Mice
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    Chapter 14 Biomarkers for Muscle Disease Gene Therapy
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    Chapter 15 Large-Scale Clinical Manufacturing of AAV Vectors for Systemic Muscle Gene Therapy
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    Chapter 16 Genome Editing for Muscle Gene Therapy
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    Chapter 17 Considerations on Preclinical Neuromuscular Disease Gene Therapy Studies
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    Chapter 18 Gene Replacement Therapy for Duchenne Muscular Dystrophy
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    Chapter 19 Recent Advances in AON-Mediated Exon-Skipping Therapy for Duchenne Muscular Dystrophy
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    Chapter 20 AAV-Mediated Exon Skipping for Duchenne Muscular Dystrophy
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    Chapter 21 Alternate Translational Initiation of Dystrophin: A Novel Therapeutic Approach
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    Chapter 22 Genome Editing for Duchenne Muscular Dystrophy
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    Chapter 23 Sarcolipin Knockdown Therapy for Duchenne Muscular Dystrophy
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    Chapter 24 Gene Therapy for Central Nervous System in Duchenne Muscular Dystrophy
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    Chapter 25 Therapeutic Approaches for Dysferlinopathy in Animal Models
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    Chapter 26 Muscle Cell Membrane Repair and Therapeutic Implications
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    Chapter 27 Dystroglycanopathy Gene Therapy: Unlocking the Potential of Genetic Engineering
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    Chapter 28 RNAi Therapy for Dominant Muscular Dystrophies and Other Myopathies
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    Chapter 29 Gene Therapy for Facioscapulohumeral Muscular Dystrophy (FSHD)
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    Chapter 30 Gene Therapy and Gene Editing for Myotonic Dystrophy
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    Chapter 31 Gene Therapy for Oculopharyngeal Muscular Dystrophy
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    Chapter 32 Gene Therapy for X-Linked Myotubular Myopathy
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    Chapter 33 Preclinical Gene Therapy Studies for Metabolic Myopathy
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    Chapter 34 Elimination of Mutant Mitochondrial DNA in Mitochondrial Myopathies Using Gene-Editing Enzymes
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    Chapter 35 Gene Therapy for CMT Inherited Neuropathy
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    Chapter 36 Muscle as a Potent Target in Vaccination
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    Chapter 37 Patient and Family Perspective on Muscle Gene Therapy
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    Chapter 38 Design of Clinical Trials for Gene Therapy in Muscular Dystrophy
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    Chapter 39 Path to Clinical Trials: Trial Design, Development of the Clinical Product, and Safety Concerns in the Implementation of Clinical Trials
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    Chapter 40 Muscle MRI as an Endpoint in Clinical Trials
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    Chapter 41 Gene Therapy Clinical Trials for Duchenne and Limb Girdle Muscular Dystrophies: Lessons Learned
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    Chapter 42 Duchenne Muscular Dystrophy Exon-Skipping Trials
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    Chapter 43 What We Have Learned from 10 Years of DMD Exon-Skipping Trials
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    Chapter 44 Clinical Gene Therapy Trials for Pompe Disease
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    Chapter 45 Muscle-Directed Gene Therapy for Alpha-1 Antitrypsin Deficiency
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Title
Muscle Gene Therapy
Published by
Springer International Publishing, March 2019
DOI 10.1007/978-3-030-03095-7
ISBNs
978-3-03-003094-0, 978-3-03-003095-7
Editors

Duan, Dongsheng, Mendell, Jerry R.

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X Demographics

X Demographics

The data shown below were collected from the profiles of 5 X users who shared this research output. Click here to find out more about how the information was compiled.
 Mendeley readers

Mendeley readers

The data shown below were compiled from readership statistics for 18 Mendeley readers of this research output. Click here to see the associated Mendeley record.

Geographical breakdown

Country Count As %
Unknown 18 100%

Demographic breakdown

Readers by professional status Count As %
Student > Master 3 17%
Researcher 3 17%
Student > Ph. D. Student 2 11%
Student > Bachelor 2 11%
Student > Doctoral Student 1 6%
Other 1 6%
Unknown 6 33%
Readers by discipline Count As %
Biochemistry, Genetics and Molecular Biology 7 39%
Engineering 2 11%
Chemical Engineering 1 6%
Neuroscience 1 6%
Nursing and Health Professions 1 6%
Other 0 0%
Unknown 6 33%

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