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Muscular Dystrophy Therapeutics

Overview of attention for book
Cover of 'Muscular Dystrophy Therapeutics'

Table of Contents

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    Book Overview
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    Chapter 1 Current Strategies of Muscular Dystrophy Therapeutics: An Overview
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    Chapter 2 Viltolarsen: From Preclinical Studies to FDA Approval
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    Chapter 3 Rapid Freezing of Skeletal and Cardiac Muscles Using Isopentane Cooled with Liquid Nitrogen and Tragacanth Gum for Histological, Genetic, and Protein Expression Studies
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    Chapter 4 Cardiac and Skeletal Muscle Pathology in the D2/mdx Mouse Model and Caveats Associated with the Quantification of Utrophin
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    Chapter 5 Physiological Assessment of Muscle, Heart, and Whole Body Function in the Canine Model of Duchenne Muscular Dystrophy
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    Chapter 6 Restoring Dystrophin Expression by Skipping Exons 6 and 8 in Neonatal Dystrophic Dogs
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    Chapter 7 Restoring Dystrophin Expression with Exon 44 and 53 Skipping in the DMD Gene in Immortalized Myotubes
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    Chapter 8 Restoring Dystrophin Expression with Duchenne Muscular Dystrophy Exon 45 Skipping in Induced Pluripotent Stem Cell-Derived Cardiomyocytes
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    Chapter 9 Quantitative Evaluation of Exon Skipping in Urine-Derived Cells for Duchenne Muscular Dystrophy
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    Chapter 10 Use of Glycine to Augment Exon Skipping and Cell Therapies for Duchenne Muscular Dystrophy
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    Chapter 11 Morpholino-Mediated Exons 28–29 Skipping of Dysferlin and Characterization of Multiexon-skipped Dysferlin using RT-PCR, Immunoblotting, and Membrane Wounding Assay
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    Chapter 12 Knocking Down DUX4 in Immortalized Facioscapulohumeral Muscular Dystrophy Patient-Derived Muscle Cells
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    Chapter 13 Peptide-Conjugated PMOs for the Treatment of Myotonic Dystrophy
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    Chapter 14 Developing Therapeutic Splice-Correcting Antisense Oligomers for Adult-Onset Pompe Disease with c.-32-13T>G Mutation
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    Chapter 15 Molecular and Biochemical Assessment of Gene Therapy in the Canine Model of Duchenne Muscular Dystrophy
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    Chapter 16 Histological Assessment of Gene Therapy in the Canine DMD Model
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    Chapter 17 MRI Evaluation of Gene Therapy in the Canine Model of Duchenne Muscular Dystrophy
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    Chapter 18 Assessment of the Gene Therapy Immune Response in the Canine Muscular Dystrophy Model
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    Chapter 19 Use of Mesenchymal Stem Cells to Enhance the Efficacy of Gene Therapy
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    Chapter 20 Exon-Skipping for a Pathogenic COL6A1 Variant in Ullrich Congenital Muscular Dystrophy
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    Chapter 21 CRISPR-Cas9 Correction of Duchenne Muscular Dystrophy in Mice by a Self-Complementary AAV Delivery System
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    Chapter 22 Preparation of NanoMEDIC Extracellular Vesicles to Deliver CRISPR-Cas9 Ribonucleoproteins for Genomic Exon Skipping
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    Chapter 23 Restoration of Dystrophin Expression in Mdx-Derived Muscle Progenitor Cells Using CRISPR/Cas9 System and Homology-Directed Repair Technology
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    Chapter 24 Effects of Glucocorticoids in Murine Models of Duchenne and Limb-Girdle Muscular Dystrophy
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    Chapter 25 High-Throughput Screening to Identify Modulators of Sarcospan
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    Chapter 26 Identifying FDA-Approved Drugs that Upregulate Utrophin A as a Therapeutic Strategy for Duchenne Muscular Dystrophy
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    Chapter 27 Monitoring Plasma Membrane Injury-Triggered Endocytosis at Single-Cell and Single-Vesicle Resolution
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    Chapter 28 Evaluation of hiPSC-Derived Muscle Progenitor Cell Transplantation in a Mouse Duchenne Muscular Dystrophy Model
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    Chapter 29 Quantification of Muscle Satellite Stem Cell Divisions by High-Content Analysis
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    Chapter 30 Systemic Delivery of a Monoclonal Antibody to Immunologically Block Myostatin in the A17 Mouse Model of OPMD
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    Chapter 31 Correction to: Cardiac and Skeletal Muscle Pathology in the D2/mdx Mouse Model and Caveats Associated with the Quantification of Utrophin
Attention for Chapter 11: Morpholino-Mediated Exons 28–29 Skipping of Dysferlin and Characterization of Multiexon-skipped Dysferlin using RT-PCR, Immunoblotting, and Membrane Wounding Assay
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Chapter title
Morpholino-Mediated Exons 28–29 Skipping of Dysferlin and Characterization of Multiexon-skipped Dysferlin using RT-PCR, Immunoblotting, and Membrane Wounding Assay
Chapter number 11
Book title
Muscular Dystrophy Therapeutics
Published by
Humana, New York, NY, November 2022
DOI 10.1007/978-1-0716-2772-3_11
Pubmed ID
36401031
Book ISBNs
978-1-07-162771-6, 978-1-07-162772-3
Authors

Anwar, Saeed, Yokota, Toshifumi

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The data shown below were collected from the profiles of 4 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 3 Mendeley readers of this research output. Click here to see the associated Mendeley record.

Geographical breakdown

Country Count As %
Unknown 3 100%

Demographic breakdown

Readers by professional status Count As %
Student > Bachelor 1 33%
Other 1 33%
Unknown 1 33%
Readers by discipline Count As %
Biochemistry, Genetics and Molecular Biology 1 33%
Agricultural and Biological Sciences 1 33%
Unknown 1 33%

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