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...
Gespeichert in:
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| Format: | Elektronisch E-Book |
| Sprache: | English |
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New York, NY
Springer New York
2018
|
| Ausgabe: | 1st ed. 2018 |
| Schriftenreihe: | Methods in Molecular Biology
1828 |
| Schlagworte: | |
| Online-Zugang: | UBR01 TUM01 URL des Erstveröffentlichers |
| Zusammenfassung: | 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 series format, chapters include introductions to their respective topics, lists of the necessary materials and reagents, step-by-step, readily reproducible laboratory protocols, and tips on troubleshooting and avoiding known pitfalls. Authoritative and cutting-edge, Exon Skipping and Inclusion Therapies: Methods and Protocols aims to help researchers initiate the development of next-generation therapies |
| Beschreibung: | 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 -- - 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 -- 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 -- - Systemic Intravenous Administration of Antisense Therapeutics for Combinatorial 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 -- - 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. |
| Beschreibung: | 1 Online-Ressource (XV, 569 Seiten) Illustrationen |
| ISBN: | 9781493986514 |
| DOI: | 10.1007/978-1-4939-8651-4 |
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| 500 | |a 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 -- | ||
| 500 | |a - 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 -- 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 -- | ||
| 500 | |a - Systemic Intravenous Administration of Antisense Therapeutics for Combinatorial 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 -- | ||
| 500 | |a - 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. | ||
| 520 | |a 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 series format, chapters include introductions to their respective topics, lists of the necessary materials and reagents, step-by-step, readily reproducible laboratory protocols, and tips on troubleshooting and avoiding known pitfalls. Authoritative and cutting-edge, Exon Skipping and Inclusion Therapies: Methods and Protocols aims to help researchers initiate the development of next-generation therapies | ||
| 650 | 4 | |a Human genetics | |
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| 700 | 1 | |a Maruyama, Rika |d ca. 20./21. Jh. |0 (DE-588)1246944286 |4 edt | |
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| dewey-full | 599.935 611.01816 |
| dewey-hundreds | 500 - Natural sciences and mathematics 600 - Technology (Applied sciences) |
| dewey-ones | 599 - Mammalia 611 - Human anatomy, cytology, histology |
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| discipline | Biologie Medizin |
| discipline_str_mv | Biologie Medizin |
| doi_str_mv | 10.1007/978-1-4939-8651-4 |
| edition | 1st ed. 2018 |
| format | Electronic eBook |
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| id | DE-604.BV047624031 |
| illustrated | Not Illustrated |
| index_date | 2024-07-03T18:43:42Z |
| indexdate | 2024-07-10T09:17:30Z |
| institution | BVB |
| isbn | 9781493986514 |
| language | English |
| oai_aleph_id | oai:aleph.bib-bvb.de:BVB01-033008592 |
| oclc_num | 1289772955 |
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| owner | DE-355 DE-BY-UBR DE-91 DE-BY-TUM |
| owner_facet | DE-355 DE-BY-UBR DE-91 DE-BY-TUM |
| physical | 1 Online-Ressource (XV, 569 Seiten) Illustrationen |
| psigel | ZDB-2-PRO |
| publishDate | 2018 |
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| publisher | Springer New York |
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| spelling | Exon skipping and inclusion therapies methods and protocols edited by Toshifumi Yokota, Rika Maruyama 1st ed. 2018 New York, NY Springer New York 2018 1 Online-Ressource (XV, 569 Seiten) Illustrationen txt rdacontent c rdamedia cr rdacarrier Methods in Molecular Biology 1828 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 -- - 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 -- 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 -- - Systemic Intravenous Administration of Antisense Therapeutics for Combinatorial 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 -- - 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. 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 series format, chapters include introductions to their respective topics, lists of the necessary materials and reagents, step-by-step, readily reproducible laboratory protocols, and tips on troubleshooting and avoiding known pitfalls. Authoritative and cutting-edge, Exon Skipping and Inclusion Therapies: Methods and Protocols aims to help researchers initiate the development of next-generation therapies Human genetics Yokota, Toshifumi ca. 20./21. Jh. (DE-588)1246943980 edt Maruyama, Rika ca. 20./21. Jh. (DE-588)1246944286 edt Erscheint auch als Druck-Ausgabe 978-1-4939-8650-7 Erscheint auch als Druck-Ausgabe 978-1-4939-8652-1 Erscheint auch als Druck-Ausgabe 978-1-4939-9354-3 https://doi.org/10.1007/978-1-4939-8651-4 Verlag URL des Erstveröffentlichers Volltext |
| spellingShingle | Exon skipping and inclusion therapies methods and protocols Human genetics |
| title | Exon skipping and inclusion therapies methods and protocols |
| title_auth | Exon skipping and inclusion therapies methods and protocols |
| title_exact_search | Exon skipping and inclusion therapies methods and protocols |
| title_exact_search_txtP | Exon skipping and inclusion therapies methods and protocols |
| title_full | Exon skipping and inclusion therapies methods and protocols edited by Toshifumi Yokota, Rika Maruyama |
| title_fullStr | Exon skipping and inclusion therapies methods and protocols edited by Toshifumi Yokota, Rika Maruyama |
| title_full_unstemmed | Exon skipping and inclusion therapies methods and protocols edited by Toshifumi Yokota, Rika Maruyama |
| title_short | Exon skipping and inclusion therapies |
| title_sort | exon skipping and inclusion therapies methods and protocols |
| title_sub | methods and protocols |
| topic | Human genetics |
| topic_facet | Human genetics |
| url | https://doi.org/10.1007/978-1-4939-8651-4 |
| work_keys_str_mv | AT yokotatoshifumi exonskippingandinclusiontherapiesmethodsandprotocols AT maruyamarika exonskippingandinclusiontherapiesmethodsandprotocols |