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Exon Skipping and Inclusion Therapies

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Cover of 'Exon Skipping and Inclusion Therapies'

Table of Contents

  1. Altmetric Badge
    Book Overview
  2. Altmetric Badge
    Chapter 1 Invention and Early History of Exon Skipping and Splice Modulation
  3. Altmetric Badge
    Chapter 2 An Overview of Recent Advances and Clinical Applications of Exon Skipping and Splice Modulation for Muscular Dystrophy and Various Genetic Diseases
  4. Altmetric Badge
    Chapter 3 Recent Advances and Clinical Applications of Exon Inclusion for Spinal Muscular Atrophy
  5. Altmetric Badge
    Chapter 4 Nusinersen in the Treatment of Spinal Muscular Atrophy
  6. Altmetric Badge
    Chapter 5 Tips to Design Effective Splice-Switching Antisense Oligonucleotides for Exon Skipping and Exon Inclusion
  7. Altmetric Badge
    Chapter 6 Antisense Oligonucleotide Targeting of 3’-UTR of mRNA for Expression Knockdown
  8. Altmetric Badge
    Chapter 7 Quantitative Evaluation of Exon Skipping in Immortalized Muscle Cells In Vitro
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    Chapter 8 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
  10. Altmetric Badge
    Chapter 9 In Vitro Multiexon Skipping by Antisense PMOs in Dystrophic Dog and Exon 7-Deleted DMD Patient
  11. Altmetric Badge
    Chapter 10 Creation of DMD Muscle Cell Model Using CRISPR-Cas9 Genome Editing to Test the Efficacy of Antisense-Mediated Exon Skipping
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    Chapter 11 In Vitro Evaluation of Exon Skipping in Disease-Specific iPSC-Derived Myocytes
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    Chapter 12 Restoration of Dystrophin Protein Expression by Exon Skipping Utilizing CRISPR-Cas9 in Myoblasts Derived from DMD Patient iPS Cells
  14. Altmetric Badge
    Chapter 13 Skipping of Duplicated Dystrophin Exons: In Vitro Induction and Assessment
  15. Altmetric Badge
    Chapter 14 In Vivo Evaluation of Dystrophin Exon Skipping in mdx Mice
  16. Altmetric Badge
    Chapter 15 Exon 51 Skipping Quantification by Digital Droplet PCR in del52hDMD/mdx Mice
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    Chapter 16 Systemic Injection of Peptide-PMOs into Humanized DMD Mice and Evaluation by RT-PCR and ELISA
  18. Altmetric Badge
    Chapter 17 In Vivo Evaluation of Single-Exon and Multiexon Skipping in mdx52 Mice
  19. Altmetric Badge
    Chapter 18 A Novel Zebrafish Model for Assessing In Vivo Delivery of Morpholino Oligomers
  20. Altmetric Badge
    Chapter 19 Validation and Detection of Exon Skipping Boosters in DMD Patient Cell Models and mdx Mouse
  21. Altmetric Badge
    Chapter 20 Use of Glucose–Fructose to Enhance the Exon Skipping Efficacy
  22. Altmetric Badge
    Chapter 21 Systemic Intravenous Administration of Antisense Therapeutics for Combinatorial Dystrophin and Myostatin Exon Splice Modulation
  23. Altmetric Badge
    Chapter 22 The Assembly of Fluorescently Labeled Peptide–Oligonucleotide Conjugates via Orthogonal Ligation Strategies
  24. Altmetric Badge
    Chapter 23 In Vivo Evaluation of Multiple Exon Skipping with Peptide-PMOs in Cardiac and Skeletal Muscles in Dystrophic Dogs
  25. Altmetric Badge
    Chapter 24 Use of Tricyclo-DNA Antisense Oligonucleotides for Exon Skipping
  26. Altmetric Badge
    Chapter 25 Optimization of 2′,4′-BNA/LNA-Based Oligonucleotides for Splicing Modulation In Vitro
  27. Altmetric Badge
    Chapter 26 Pre-mRNA Splicing Modulation by Antisense Oligonucleotides
  28. Altmetric Badge
    Chapter 27 In Vitro Evaluation of Antisense-Mediated Exon Inclusion for Spinal Muscular Atrophy
  29. Altmetric Badge
    Chapter 28 Systemic and ICV Injections of Antisense Oligos into SMA Mice and Evaluation
  30. Altmetric Badge
    Chapter 29 Morpholino-Mediated Exon Inclusion for SMA
  31. Altmetric Badge
    Chapter 30 Exon Skipping by Ultrasound-Enhanced Delivery of Morpholino with Bubble Liposomes for Myotonic Dystrophy Model Mice
  32. Altmetric Badge
    Chapter 31 Dysferlin Exon 32 Skipping in Patient Cells
  33. Altmetric Badge
    Chapter 32 Morpholino-Mediated Exon Skipping Targeting Human ACVR1/ALK2 for Fibrodysplasia Ossificans Progressiva
  34. Altmetric Badge
    Chapter 33 Exon Skipping of FcεRIβ for Allergic Diseases
  35. Altmetric Badge
    Chapter 34 Antisense Oligonucleotide Design and Evaluation of Splice-Modulating Properties Using Cell-Based Assays
  36. Altmetric Badge
    Chapter 35 Antisense-Mediated Splice Modulation to Reframe Transcripts
  37. Altmetric Badge
    Chapter 36 Exon Skipping Using Antisense Oligonucleotides for Laminin-Alpha2-Deficient Muscular Dystrophy
Attention for Chapter 6: Antisense Oligonucleotide Targeting of 3’-UTR of mRNA for Expression Knockdown
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Chapter title
Antisense Oligonucleotide Targeting of 3’-UTR of mRNA for Expression Knockdown
Chapter number 6
Book title
Exon Skipping and Inclusion Therapies
Published in
Methods in molecular biology, September 2018
DOI 10.1007/978-1-4939-8651-4_6
Pubmed ID
Book ISBNs
978-1-4939-8650-7, 978-1-4939-8651-4
Authors

Golnoush Golshirazi, Lukasz Ciszewski, Ngoc Lu-Nguyen, Linda Popplewell

Abstract

With the recent conditional approval of an antisense oligonucleotide (AON) that restores the reading frame of DMD transcript in a subset of Duchenne muscular dystrophy patients, it has been established that AONs sharing similar chemistry have clear clinical potential. Genetic diseases, such as facioscapulohumeral dystrophy (FSHD), can be the result of gain-of-function mutations. Since mRNA processing in terms of termination of transcription, its transport from the nucleus to the cytoplasm, its stability and translation efficiency are dependent on key 3'UTR elements, it follows that targeting these elements with AONs have the potential to induce gene silencing. Aberrant expression of the Double homeobox 4 (DUX4) transcription factor and the downstream consequences of such expression is the hallmark of FSHD. Here we describe the bioinformatic strategies behind the design of AONs targeting polyadenylation signals and the methodologies relevant to their in vitro screening for efficacy and safety, including analysis of expression at the transcript and protein level of the specific target and downstream genes, and measurement of the effect on the fusion index of myotubes. The targeting of permissive DUX4 and MSTN are used as examples. MSTN encodes for myostatin, a negative regulator of myogenesis; the downregulation of MSTN expression has the potential to address the muscular atrophy associated with muscular dystrophies, sarcopenia, cancer and acquired immunodeficiency syndrome.

Twitter Demographics

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

Mendeley readers

The data shown below were compiled from readership statistics for 9 Mendeley readers of this research output. Click here to see the associated Mendeley record.

Geographical breakdown

Country Count As %
Unknown 9 100%

Demographic breakdown

Readers by professional status Count As %
Student > Ph. D. Student 2 22%
Researcher 2 22%
Professor 1 11%
Student > Master 1 11%
Unknown 3 33%
Readers by discipline Count As %
Biochemistry, Genetics and Molecular Biology 3 33%
Agricultural and Biological Sciences 3 33%
Unknown 3 33%

Attention Score in Context

This research output has an Altmetric Attention Score of 3. This is our high-level measure of the quality and quantity of online attention that it has received. This Attention Score, as well as the ranking and number of research outputs shown below, was calculated when the research output was last mentioned on 05 September 2018.
All research outputs
#6,904,212
of 13,497,563 outputs
Outputs from Methods in molecular biology
#1,736
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Outputs of similar age
#109,046
of 265,609 outputs
Outputs of similar age from Methods in molecular biology
#1
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Altmetric has tracked 13,497,563 research outputs across all sources so far. This one is in the 48th percentile – i.e., 48% of other outputs scored the same or lower than it.
So far Altmetric has tracked 8,491 research outputs from this source. They receive a mean Attention Score of 2.1. This one has done well, scoring higher than 79% of its peers.
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