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| 008 | 230516s2018 xxu| o |||| 0|eng d | ||
| 020 | _a9781493986514 | ||
| 024 | 7 |
_a10.1007/978-1-4939-8651-4 _2doi |
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| 040 |
_aES-MaUEC _bspa _cES-MaUEC _dES-MaUEC |
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_aRB155.8 _b2018 EB |
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| 245 | 0 | 0 |
_aExon Skipping and Inclusion Therapies : _bMethods and Protocols _cedited by Toshifumi Yokota, Rika Maruyama |
| 250 | _a1st edition 2018 | ||
| 264 | 1 |
_aNew York, NY _bSpringer International Publishing _c2018 |
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| 300 |
_a1 recurso en línea (XV, 569 páginas) _b103 ilustraciones, 77 ilustraciones a color |
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| 336 |
_atexto _btxt _2rdacontent |
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| 337 |
_aelectrónico _bc _2rdamedia |
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| 338 |
_arecurso electrónico _bcr _2rdacarrier |
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| 347 |
_aarchivo de texto _bPDF |
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| 490 | 0 |
_aMethods in Molecular Biology _x1940-6029 _v1828 |
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| 505 | 0 | _aInvention 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. . | |
| 520 | _aThis 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. | ||
| 988 | _aSpringer_Protocols_2018 | ||
| 650 | 7 |
_2embne _9144879 _aGenética médica |
|
| 650 | 7 |
_2embne _9169472 _aMúsculos _xEnfermedades |
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| 650 | 7 |
_2embne _9150702 _aTerapia genética |
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| 776 | 0 | 8 |
_iPrinted edition: _z9781493986507 |
| 776 | 0 | 8 |
_iPrinted edition: _z9781493986521 |
| 776 | 0 | 8 |
_iPrinted edition: _z9781493993543 |
| 856 | 4 | 0 |
_uhttps://go.openathens.net/redirector/universidadeuropea.es?url=https://doi.org/10.1007/978-1-4939-8651-4 _zAcceso a este recurso digital (usuarios Universidad Europea de Madrid) |
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| 998 |
_b05/2023 _dz _eIG _zSI |
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