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04396nam a22003015i 4500 |
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20201005083402.0 |
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cr nn 008mamaa |
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180831s2018 xxu| s |||| 0|eng d |
| 020 |
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|a 9781493986514
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| 024 |
7 |
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|a 10.1007/978-1-4939-8651-4
|2 doi
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| 040 |
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|a Sistema de Bibliotecas del Tecnológico de Costa Rica
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| 245 |
1 |
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|a Exon Skipping and Inclusion Therapies :
|b Methods and Protocols /
|c edited by Toshifumi Yokota, Rika Maruyama.
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| 250 |
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|a 1st ed. 2018.
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| 260 |
# |
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|a New York, NY :
|b Springer New York :
|b Imprint: Humana,
|c 2018.
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| 300 |
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|a XV, 569 p. 103 illus., 77 illus. in color. :
|b online resource.
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| 336 |
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|a text
|b txt
|2 rdacontent
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| 337 |
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|a computer
|b c
|2 rdamedia
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| 338 |
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|a online resource
|b cr
|2 rdacarrier
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| 490 |
1 |
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|a Methods in Molecular Biology,
|v 1828
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| 505 |
0 |
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|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 -- 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. .
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| 650 |
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|a Human genetics.
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| 650 |
1 |
4 |
|a Human Genetics.
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| 700 |
1 |
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|a Yokota, Toshifumi.
|e editor.
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| 700 |
1 |
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|a Maruyama, Rika.
|e editor.
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| 710 |
2 |
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|a SpringerLink (Online service)
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| 773 |
0 |
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|t Springer eBooks
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| 900 |
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|a Libro descargado a ALEPH en bloque (proveniente de proveedor)
|