| Description |
1 online resource (xv, 569 pages) : illustrations (some color) |
| Series |
Methods in molecular biology, 1940-6029 ; v. 1828 |
|
Methods in molecular biology (Clifton, N.J.) ; v. 1828. 1064-3745
|
| Contents |
880-01 Invention and early history of exon skipping and splice modulation / Kenji Rowel Q. Lim and Toshifumi Yokota -- Overview of recent advances and clinical applications of exon skipping and splice modulation for muscular dystrophy and various genetic diseases / Merryl Rodrigues and Toshifumi Yokota -- Recent advances and clinical applications of exon inclusion for spinal muscular atrophy / Hae-Won Son and Toshifumi Yokota -- Nusinersen in the treatment of spinal muscular atrophy / Kara Goodkey, Tejal Aslesh, Rika Maruyama, and Toshifumi Yokota -- Tips to design effective splice-switching antisense oligonucleotides for exon skipping and exon inclusion / Rika Maruyama and Toshifumi Yokota -- Antisense oligonucleotide targeting of 3'-UTR of mRNA for expression knockdown / Golnoush Golshirazi, Lukasz Ciszewski, Ngoc Lu-Nguyen, and Linda Popplewell -- Quantitative evaluation of exon skipping in immortalized muscle cells in vitro / Kenji Rowel Q. Lim and Toshifumi Yokota -- 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, Shinichi Takeda, and Toshifumi Yokota -- In vitro multiexon skipping by antisense PMOs in dystrophic dog and exon 7-deleted DMD patient / Akinori Nakamura, Yoshitsugu Aoki, Maria Tsoumpra, Toshifumi Yokota, and Shinichi Takeda -- Creation of DMD muscle cell model using CRISPR-Cas9 genome editing to test the efficacy of antisense-mediated exon skipping / Rika Maruyama and Toshifumi Yokota -- In vitro evaluation of exon skipping in disease-specific iPSC-derived myocytes / Mingming Zhao, Emi Shoji, and Hidetoshi Sakurai -- 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, and Akitsu Hotta -- Skipping of duplicated dystrophin exons : in vitro induction and assessment / Kane Greer, Sue Fletcher, and Steve D. Wilton -- In vivo evaluation of dystrophin exon skipping in mdx mice / Bo Wu, Mingxing Wang, Sapana Shah, and Qi Long Lu -- Exon 51 skipping quantification by digital droplet PCR in del52hDMD/mdx Mice / Monika Hiller, Pietro Spitali, Nicole Datson, and Annemieke Aartsma-Rus -- Systemic injection of peptide-PMOs into humanized DMD mice and evaluation by RT-PCR and ELISA / Dyanna Melo, Rika Maruyama, and Toshifumi Yokota -- In vivo evaluation of single-exon and multiexon skipping in mdx52 mice / Yoshitaka Mizobe, Shouta Miyatake, Hotake Takizawa, Yuko Hara, Toshifumi Yokota, Akinori Nakamura, ShinIchi Takeda, and Yoshitsugu Aoki -- Novel zebrafish model for assessing In vivo delivery of morpholino oligomers / Joseph Kim, Karl Clark, Carrie Barton, Robert Tanguay, and Hong Moulton -- Validation and detection of exon skipping boosters in DMD patient cell models and mdx mouse / Florian Barthelemy, Dereck Wang, Stanley F. Nelson, and M. Carrie Miceli -- Use of glucose-fructose to enhance the exon skipping efficacy / Gang Han, Xianjun Gao, and Haifang Yin -- Systemic intravenous administration of antisense therapeutics for combinatorial dystrophin and myostatin exon splice modulation / Ngoc Lu-Nguyen, George Dickson, and Alberto Malerba -- Assembly of fluorescently labeled peptide-oligonucleotide conjugates via orthogonal ligation strategies / John Karas, Bradley J. Turner, and Fazel Shabanpoor -- In vivo evaluation of multiple exon skipping with peptide-PMOs in cardiac and skeletal muscles in dystrophic dogs / Rika Maruyama, Yoshitsugu Aoki, Shinichi Takeda, and Toshifumi Yokota -- Use of tricyclo-DNA antisense oligonucleotides for exon skipping / Karima Relizani and Aurelie Goyenvalle -- Optimization of 2',4'-BNA/LNA-based oligonucleotides for splicing modulation in vitro / Takenori Shimo and Satoshi Obika -- Pre-mRNA splicing modulation by antisense oligonucleotides / Natalia N. Singh, Diou Luo, and Ravindra N. Singh -- In vitro evaluation of antisense-mediated exon inclusion for spinal muscular atrophy / Aleksander Touznik, Rika Maruyama, and Toshifumi Yokota -- Systemic and ICV injections of antisense oligos into SMA mice and evaluation / Tejal Aslesh, Rika Maruyama, and Toshifumi Yokota -- Morpholino-mediated exon inclusion for SMA / Haiyan Zhou and Francesco Muntoni -- Exon skipping by ultrasound-enhanced delivery of morpholino with bubble liposomes for myotonic dystrophy model mice / Yoichi Negishi, Yoko Endo-Takahashi, and Shoichi Ishiura -- Dysferlin exon 32 skipping in patient cells / Florian Barthélémy, Sébastien Courrier, Nicolas Lévy, Martin Krahn, and Marc Bartoli -- Morpholino-mediated exon skipping targeting human ACVR1/ALK2 for fibrodysplasia ossificans progressiva / Rika Maruyama and Toshifumi Yokota -- Exon skipping of Fc?RI? for allergic diseases / Greer K. Arthur and Glenn Cruse -- Antisense oligonucleotide design and evaluation of splice-modulating properties using cell-based assays / Ralph Slijkerman, Hannie Kremer, and Erwin van Wijk -- Antisense-mediated splice modulation to reframe transcripts / Matthias Titeux, Sandrina Turczynski, Nathalie Pironon, and Alain Hovnanian -- Exon skipping using antisense oligonucleotides for laminin-alpha2-deficient muscular dystrophy / Yuko Hara, Yoshitaka Mizobe, Shouta Miyatake, Hotake Takizawa, Tetsuya Nagata, Toshifumi Yokota, Shinichi Takeda, and Yoshitsugu Aoki |
|
880-01/(S Invention and early history of exon skipping and splice modulation / Kenji Rowel Q. Lim and Toshifumi Yokota -- Overview of recent advances and clinical applications of exon skipping and splice modulation for muscular dystrophy and various genetic diseases / Merryl Rodrigues and Toshifumi Yokota -- Recent advances and clinical applications of exon inclusion for spinal muscular atrophy / Hae-Won Son and Toshifumi Yokota -- Nusinersen in the treatment of spinal muscular atrophy / Kara Goodkey, Tejal Aslesh, Rika Maruyama, and Toshifumi Yokota -- Tips to design effective splice-switching antisense oligonucleotides for exon skipping and exon inclusion / Rika Maruyama and Toshifumi Yokota -- Antisense oligonucleotide targeting of 3'-UTR of mRNA for expression knockdown / Golnoush Golshirazi, Lukasz Ciszewski, Ngoc Lu-Nguyen, and Linda Popplewell -- Quantitative evaluation of exon skipping in immortalized muscle cells in vitro / Kenji Rowel Q. Lim and Toshifumi Yokota -- 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, Shinichi Takeda, and Toshifumi Yokota -- In vitro multiexon skipping by antisense PMOs in dystrophic dog and exon 7-deleted DMD patient / Akinori Nakamura, Yoshitsugu Aoki, Maria Tsoumpra, Toshifumi Yokota, and Shinichi Takeda -- Creation of DMD muscle cell model using CRISPR-Cas9 genome editing to test the efficacy of antisense-mediated exon skipping / Rika Maruyama and Toshifumi Yokota -- In vitro evaluation of exon skipping in disease-specific iPSC-derived myocytes / Mingming Zhao, Emi Shoji, and Hidetoshi Sakurai -- 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, and Akitsu Hotta -- Skipping of duplicated dystrophin exons : in vitro induction and assessment / Kane Greer, Sue Fletcher, and Steve D. Wilton -- In vivo evaluation of dystrophin exon skipping in mdx mice / Bo Wu, Mingxing Wang, Sapana Shah, and Qi Long Lu -- Exon 51 skipping quantification by digital droplet PCR in del52hDMD/mdx Mice / Monika Hiller, Pietro Spitali, Nicole Datson, and Annemieke Aartsma-Rus -- Systemic injection of peptide-PMOs into humanized DMD mice and evaluation by RT-PCR and ELISA / Dyanna Melo, Rika Maruyama, and Toshifumi Yokota -- In vivo evaluation of single-exon and multiexon skipping in mdx52 mice / Yoshitaka Mizobe, Shouta Miyatake, Hotake Takizawa, Yuko Hara, Toshifumi Yokota, Akinori Nakamura, ShinIchi Takeda, and Yoshitsugu Aoki -- Novel zebrafish model for assessing In vivo delivery of morpholino oligomers / Joseph Kim, Karl Clark, Carrie Barton, Robert Tanguay, and Hong Moulton -- Validation and detection of exon skipping boosters in DMD patient cell models and mdx mouse / Florian Barthelemy, Dereck Wang, Stanley F. Nelson, and M. Carrie Miceli -- Use of glucose-fructose to enhance the exon skipping efficacy / Gang Han, Xianjun Gao, and Haifang Yin -- Systemic intravenous administration of antisense therapeutics for combinatorial dystrophin and myostatin exon splice modulation / Ngoc Lu-Nguyen, George Dickson, and Alberto Malerba -- Assembly of fluorescently labeled peptide-oligonucleotide conjugates via orthogonal ligation strategies / John Karas, Bradley J. Turner, and Fazel Shabanpoor -- In vivo evaluation of multiple exon skipping with peptide-PMOs in cardiac and skeletal muscles in dystrophic dogs / Rika Maruyama, Yoshitsugu Aoki, Shinichi Takeda, and Toshifumi Yokota -- Use of tricyclo-DNA antisense oligonucleotides for exon skipping / Karima Relizani and Aurelie Goyenvalle -- Optimization of 2',4'-BNA/LNA-based oligonucleotides for splicing modulation in vitro / Takenori Shimo and Satoshi Obika -- Pre-mRNA splicing modulation by antisense oligonucleotides / Natalia N. Singh, Diou Luo, and Ravindra N. Singh -- In vitro evaluation of antisense-mediated exon inclusion for spinal muscular atrophy / Aleksander Touznik, Rika Maruyama, and Toshifumi Yokota -- Systemic and ICV injections of antisense oligos into SMA mice and evaluation / Tejal Aslesh, Rika Maruyama, and Toshifumi Yokota -- Morpholino-mediated exon inclusion for SMA / Haiyan Zhou and Francesco Muntoni -- Exon skipping by ultrasound-enhanced delivery of morpholino with bubble liposomes for myotonic dystrophy model mice / Yoichi Negishi, Yoko Endo-Takahashi, and Shoichi Ishiura -- Dysferlin exon 32 skipping in patient cells / Florian Barthélémy, Sébastien Courrier, Nicolas Lévy, Martin Krahn, and Marc Bartoli -- Morpholino-mediated exon skipping targeting human ACVR1/ALK2 for fibrodysplasia ossificans progressiva / Rika Maruyama and Toshifumi Yokota -- Exon skipping of FcεRIβ for allergic diseases / Greer K. Arthur and Glenn Cruse -- Antisense oligonucleotide design and evaluation of splice-modulating properties using cell-based assays / Ralph Slijkerman, Hannie Kremer, and Erwin van Wijk -- Antisense-mediated splice modulation to reframe transcripts / Matthias Titeux, Sandrina Turczynski, Nathalie Pironon, and Alain Hovnanian -- Exon skipping using antisense oligonucleotides for laminin-alpha2-deficient muscular dystrophy / Yuko Hara, Yoshitaka Mizobe, Shouta Miyatake, Hotake Takizawa, Tetsuya Nagata, Toshifumi Yokota, Shinichi Takeda, and Yoshitsugu Aoki |
| Bibliography |
Includes bibliographical references and index |
| Notes |
Online resource; title from PDF title page (SpringerLink, viewed September 5, 2018) |
| Subject |
Gene therapy -- Laboratory manuals
|
|
Exons (Genetics) -- Laboratory manuals
|
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Exons (Genetics)
|
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Exons
|
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Genetic Therapy -- methods
|
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Muscular Dystrophy, Duchenne -- therapy
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Muscular Atrophy, Spinal -- therapy
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Medical genetics.
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Medical -- Genetics.
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Exons (Genetics)
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Gene therapy
|
| Genre/Form |
Laboratory manuals
|
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Laboratory manuals.
|
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Manuels de laboratoire.
|
| Form |
Electronic book
|
| Author |
Yokota, Toshifumi, editor
|
|
Maruyama, Rika, editor
|
| ISBN |
9781493986514 |
|
1493986511 |
|
