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Produktbild: Exon Skipping and Inclusion Therapies
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Exon Skipping and Inclusion Therapies Methods and Protocols

Aus der Reihe Methods in Molecular Biology
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173,99 € UVP 197,99 €

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Beschreibung

Produktdetails

Einband

Gebundene Ausgabe

Erscheinungsdatum

29.07.2025

Abbildungen

XIV, 87 illus., 68 illus. in color., farbige Illustrationen, schwarz-weiss Illustrationen

Herausgeber

Yusuke Echigoya + weitere

Verlag

Springer Us

Seitenzahl

503

Maße (L/B/H)

26/18,3/3,4 cm

Gewicht

1163 g

Auflage

Second Edition 2025

Sprache

Englisch

ISBN

978-1-07-164729-5

Beschreibung

Produktdetails

Einband

Gebundene Ausgabe

Erscheinungsdatum

29.07.2025

Abbildungen

XIV, 87 illus., 68 illus. in color., farbige Illustrationen, schwarz-weiss Illustrationen

Herausgeber

Verlag

Springer Us

Seitenzahl

503

Maße (L/B/H)

26/18,3/3,4 cm

Gewicht

1163 g

Auflage

Second Edition 2025

Sprache

Englisch

ISBN

978-1-07-164729-5

Herstelleradresse

Libri GmbH
Europaallee 1
36244 Bad Hersfeld
DE

Email: GPSR Kontakt

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  • Produktbild: Exon Skipping and Inclusion Therapies

  • Progress and Future Directions in Exon Skipping and Inclusion Therapies: The Landscape of Oligonucleotide-Based Genetic Medicine.- Evolution and Breakthroughs in Exon Skipping and Splice Modulation: From Inception to Clinical Success.- An Overview of Recent Advances and Clinical Applications of Exon Skipping and Splice Modulation for Muscular Dystrophy and Various Genetic Diseases.- Milasen: The Emerging Era of Patient-Customized N-of-1 Antisense Oligonucleotides as Therapeutic Agents for Genetic Diseases.- Tips to Design Effective Splice-Switching Antisense Oligonucleotides for Exon Skipping and Exon Inclusion.- In Silico Prediction and Selection of Exon-Skipping Antisense Oligonucleotide Sequences Using eSkip-Finder.- Quantitative Evaluation of Exon Skipping in Immortalized Muscle Cells In Vitro.- Direct Reprogramming of Human DMD Fibroblasts into Myotubes for In Vitro Evaluation of Antisense-Mediated Exon Skipping and Exons 45-55 Skipping Accompanied by Rescue of Dystrophin Expression.- In Vitro Multiexon Skipping by Antisense PMOs in Dystrophic Dog and Exon 7-Deleted DMD Patient.- Creation of DMD Muscle Cell Model Using CRISPR-Cas9 Genome Editing to Test the Efficacy of Antisense-Mediated Exon Skipping.- In Vitro Evaluation of Exon Skipping in Disease Specific iPSC-Derived Myocytes.- Skipping of Duplicated Dystrophin Exons: In Vitro Induction and Assessment.- In Vivo Evaluation of Dystrophin Exon Skipping in mdx Mice.- Guide to Selection of Muscle-Homing Peptides after In Vivo Phage Display Biopanning.- Systemic Injection of Peptide-PMOs into Humanized DMD Mice and Detection by RT-PCR and ELISA.- In Vivo Evaluation of Single- and Multi-Exon Skipping in mdx52 Mice.- A Novel Zebrafish Model for Assessing In Vivo Delivery of Morpholino Oligomers.- Electrophysiological Evaluation in mdx52 Mouse Brain after Antisense-Mediated Exon 51 or 53 Skipping.- Use of Glucose/Fructose to Enhance the Exon Skipping Efficacy.- Systemic Intravenous Administration of Antisense Therapeutics for Combinatorial Dystrophin and Myostatin Exon Splice Modulation.- The Assembly of Fluorescently Labeled Peptide-Oligonucleotide Conjugates.- In Vivo Evaluation of Multiple Exon Skipping with Peptide-PMOs in Cardiac and Skeletal Muscles in Dystrophic Dogs.- Use of Tricyclo-DNA Antisense Oligonucleotides for Exon Skipping.- Optimization of 2¿,4¿-BNA/LNA-Based Oligonucleotides for Splicing Modulation In Vitro.- Pre-mRNA Splicing Modulation by Antisense Oligonucleotides.- In Vitro Evaluation of Antisense-Mediated Exon Inclusion for Spinal Muscular Atrophy.- Systemic Injection of Antisense Oligos into Spinal Muscular Atrophy (SMA) Mice and Evaluation.- Morpholino-Mediated Exon Inclusion for Spinal Muscular Atrophy (SMA).- Exon Skipping by Ultrasound-Enhanced Delivery of Morpholino with Bubble Liposomes for Myotonic Dystrophy Model Mice.- Restoration of Dysferlin after Exon 32 Skipping in Patient Cells.- Morpholino-Mediated Exon Skipping Targeting Human ACVR1/ALK2 for Fibrodysplasia Ossificans Progressiva.- Exon Skipping of FcεRIβ for Allergic Diseases.- Exon-Skipping Using Antisense Oligonucleotides for Laminin-Alpha2-Deficient Muscular Dystrophy.