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Home > Exon Skipping and Inclusion Therapies: Methods and Protocols > Quantitative Evaluation of Exon Skipping in Immortalized Muscle Cells In Vitro
Exon Skipping and Inclusion Therapies: Methods and Protocols |
Toshifumi Yokota; Rika Maruyama
Springer New York
Exon Skipping and Inclusion Therapies: Methods and Protocols
Quantitative Evaluation of Exon Skipping in Immortalized Muscle Cells In Vitro
Authors

Kenji Rowel Q. Lim; Toshifumi Yokota

ARI Id

1665140120565_5002716

Access

Not Available Free

Pages

127-139

DOI

10.1007/978-1-4939-8651-4_7

Chapter URL

https://rd.springer.com/protocol/10.1007/978-1-4939-8651-4_7

Table of Contents of Book
Showing 1 to 20 of 46 entries
Chapters/HeadingsAuthor(s)PagesInfo
Front Matter
i-xv
Front Matter
1-1
Invention and Early History of Exon Skipping and Splice Modulation
Kenji Rowel Q. Lim; Toshifumi Yokota
3-30
An Overview of Recent Advances and Clinical Applications of Exon Skipping and Splice Modulation for Muscular Dystrophy and Various Genetic Diseases
Merryl Rodrigues; Toshifumi Yokota
31-55
Recent Advances and Clinical Applications of Exon Inclusion for Spinal Muscular Atrophy
Hae-Won Son; Toshifumi Yokota
57-68
Nusinersen in the Treatment of Spinal Muscular Atrophy
Kara Goodkey; Tejal Aslesh; Rika Maruyama; Toshifumi Yokota
69-76
Front Matter
77-77
Tips to Design Effective Splice-Switching Antisense Oligonucleotides for Exon Skipping and Exon Inclusion
Rika Maruyama; Toshifumi Yokota
79-90
Antisense Oligonucleotide Targeting of 3’-UTR of mRNA for Expression Knockdown
Golnoush Golshirazi; Lukasz Ciszewski; Ngoc Lu-Nguyen; Linda Popplewell
91-124
Front Matter
125-125
Quantitative Evaluation of Exon Skipping in Immortalized Muscle Cells In Vitro
Kenji Rowel Q. Lim; Toshifumi Yokota
127-139
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
Joshua J. A. Lee; Takashi Saito; William Duddy; Shin’ichi Takeda; Toshifumi Yokota
141-150
In Vitro Multiexon Skipping by Antisense PMOs in Dystrophic Dog and Exon 7-Deleted DMD Patient
Akinori Nakamura; Yoshitsugu Aoki; Maria Tsoumpra; Toshifumi Yokota; Shin’ichi Takeda
151-163
Creation of DMD Muscle Cell Model Using CRISPR-Cas9 Genome Editing to Test the Efficacy of Antisense-Mediated Exon Skipping
Rika Maruyama; Toshifumi Yokota
165-171
In Vitro Evaluation of Exon Skipping in Disease-Specific iPSC-Derived Myocytes
Mingming Zhao; Emi Shoji; Hidetoshi Sakurai
173-189
Restoration of Dystrophin Protein Expression by Exon Skipping Utilizing CRISPR-Cas9 in Myoblasts Derived from DMD Patient iPS Cells
Masataka Ifuku; Kumiko A. Iwabuchi; Masami Tanaka; Mandy Siu Yu Lung; Akitsu Hotta
191-217
Skipping of Duplicated Dystrophin Exons: In Vitro Induction and Assessment
Kane Greer; Sue Fletcher; Steve D. Wilton
219-228
Front Matter
229-229
In Vivo Evaluation of Dystrophin Exon Skipping in mdx Mice
Bo Wu; Mingxing Wang; Sapana Shah; Qi Long Lu
231-247
Exon 51 Skipping Quantification by Digital Droplet PCR in del52hDMD/mdx Mice
Monika Hiller; Pietro Spitali; Nicole Datson; Annemieke Aartsma-Rus
249-262
Chapters/HeadingsAuthor(s)PagesInfo
Showing 1 to 20 of 46 entries
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