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E-grāmata: Muscular Dystrophy Therapeutics: Methods and Protocols

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  • Formāts: EPUB+DRM
  • Sērija : Methods in Molecular Biology 2587
  • Izdošanas datums: 18-Nov-2022
  • Izdevniecība: Springer-Verlag New York Inc.
  • Valoda: eng
  • ISBN-13: 9781071627723
  • Formāts - EPUB+DRM
  • Cena: 213,54 €*
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  • Formāts: EPUB+DRM
  • Sērija : Methods in Molecular Biology 2587
  • Izdošanas datums: 18-Nov-2022
  • Izdevniecība: Springer-Verlag New York Inc.
  • Valoda: eng
  • ISBN-13: 9781071627723

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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.
Current Strategies of Muscular Dystrophy Therapeutics: An Overview.- The
Story of Viltolarsen: From Preclinical Studies to FDA Approval.- Rapid
Freezing of Skeletal and Cardiac Muscles Using Isopentane Cooled with Liquid
Nitrogen and Tragacanth Gum for Histological, Genetic, and Protein Expression
Studies.- Cardiac and Skeletal Muscle Pathology in the D2/mdx Mouse Model and
Caveats Associated with the Quantification of Utrophin.- Physiological
Assessment of Muscle, Heart, and Whole Body Function in the Canine Model of
Duchenne Muscular Dystrophy.- Restoring Dystrophin Expression by Skipping
Exon 6 and 8 in Neonatal Dystrophic Dogs.- Restoring Dystrophin Expression
with Exon 44 and 53 Skipping in the DMD Gene in Immortalized Myotubes.-
Restoring Dystrophin Expression with Duchenne Muscular Dystrophy Exon 45
Skipping in Induced-Pluripotent Stem Cell-Derived Cardiomyocytes.-
Quantitative Evaluation of Exon Skipping in Urine-Derived Cells for Duchenne
Muscular Dystrophy.- Use of Glycine to Augment Exon Skipping and Cell
Therapies for Duchenne Muscular Dystrophy.- Morpholino-Mediated Exons 2829
Skipping in Dysferlin.- Knocking Down DUX4 in Immortalized
Facioscapulohumeral Muscular Dystrophy Patient-Derived Muscle Cells.-
Peptide-Conjugated PMOs for the Treatment of Myotonic Dystrophy.- Developing
Therapeutic Splice-Correcting Antisense Oligomers for Adult-Onset Pompe
Disease with c.-32-13T>G Mutation.- Molecular and Biochemical Assessment of
Gene Therapy in the Canine Model of Duchenne Muscular Dystrophy.-
Histological Assessment of Gene Therapy in the Canine DMD Model.- MRI
Evaluation of Gene Therapy in the Canine Model of Duchenne Muscular
Dystrophy.- Assessment of the Gene Therapy Immune Response in the Canine
Muscular Dystrophy Model.- Use of Mesenchymal Stem Cells to Enhance the
Efficacy of Gene Therapy.- Exon-Skipping for a Pathogenic COL6A1 Variant in
Ullrich CMD.- CRISPR-Cas9 Correction of Duchenne Muscular Dystrophy in Mice
by a Self-Complementary AAV Delivery System.- Preparation of NanoMEDIC
Extracellular Vesicles to Deliver CRISPR-Cas9 Ribonucleoproteins for Genomic
Exon Skipping.- Restoration of Dystrophin Expression in Mdx-Derived Muscle
Progenitor Cells Using CRISPR/Cas9 System and Homology-Directed Repair
Technology.- Effects of Glucocorticoids in Murine Models of Duchenne and
Limb-Girdle Muscular Dystrophy.- High-Throughput Screening to Identify
Modulators of Sarcospan.- Identifying FDA-Approved Drugs that Upregulate
Utrophin A as a Therapeutic Strategy for Duchenne Muscular Dystrophy.-
Monitoring Membrane Injury-Triggered Endocytosis at Single Cell and Single
Vesicle Resolution.- Evaluation of hiPSC-Derived Muscle Progenitor Cell
Transplantation in a Mouse Duchenne Muscular Dystrophy Model.- Quantification
of Muscle Satellite Stem Cell Divisions by High Content Analysis.- Systemic
Delivery of a Monoclonal Antibody to Immunologically Block Myostatin in the
A17 Mouse Model of OPMD.