Exon Skipping and Inclusion Therapies Methods and Protocols
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 serie...
| Other Authors: | , |
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| Format: | eBook |
| Language: | English |
| Published: |
New York, NY
Humana
2018, 2018
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| Edition: | 1st ed. 2018 |
| Series: | Methods in Molecular Biology
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| Subjects: | |
| Online Access: | |
| Collection: | Springer eBooks 2005- - Collection details see MPG.ReNa |
Table of Contents:
- Systemic Intravenous Administration of Antisense Therapeutics forCombinatorial Dystrophin and Myostatin Exon Splice Modulation
- The Assembly of Fluorescently Labeled Peptide-oligonucleotide Conjugates via Orthogonal Ligation Strategies
- 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 2ʹ,4ʹ-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 SMA Mice and Evaluation
- Exon Skipping using Antisense Oligonucleotides for Laminin-alpha2-deficient Muscular Dystrophy
- Exon Skipping by Ultrasound-enhanced Delivery of Morpholino with Bubble Liposomes for Myotonic Dystrophy Model Mice
- Creation of DMD Muscle Cell Model using CRISPR-Cas9 Genome Editing toTest the Efficacy of Antisense-mediated Exon Skipping
- In vitro Evaluation of Exon Skipping in Disease Specific iPSC-derived Myocytes
- Restoration of Dystrophin Protein Expression by Exon Skipping utilizing CRISPR-Cas9 in Myoblasts Derived from DMD Patient iPS Cells
- Skipping of Duplicated Dystrophin Exons: in vitro Induction and Assessment
- In Vivo Evaluation of Dystrophin Exon Skipping in mdx Mice
- Exon 51 Skipping Quantification by Digital Droplet PCR in del52hDMD/mdx Mice
- 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
- Validation and Detection of Exon Skipping Boosters in DMD Patient Cell Models and mdx Mouse
- Use of Glucose/Fructose to Enhance the Exon Skipping Efficacy
- Invention and Early History of Exon Skipping and Splice Modulation
- An Overview of Recent Advances and Clinical Applications of Exon Skipping and Splice Modulation for Muscular Dystrophy and Various Genetic Diseases
- Recent Advances and Clinical Applications of Exon Inclusion for Spinal Muscular Atrophy
- Nusinersen in the Treatment of Spinal Muscular Atrophy
- Tips to Design Effective Splice-switching Antisense Oligonucleotides for Exon Skipping and Exon Inclusion
- Antisense Oligonucleotide Targeting of 3’UTR of mRNA for Expression Knockdown
- 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-m Exon Skipping and Exons 45-55 Skipping Accompanied by Rescue of Dystrophin Expression
- In vitro Multi-exon Skipping by Antisense PMOs in Dystrophic Dog and Exon 7-Deleted DMD Patient
- Dysferlin 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
- Antisense Oligonucleotide Design and Evaluation of Splice-modulating Properties Using Cell-based Assays
- Antisense-mediated Splice Modulation to Reframe Transcripts
- Morpholino-mediated Exon Inclusion for SMA.