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Splice Switching: Methods and Protocols [Kõva köide]

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  • Formaat: Hardback, 146 pages, kõrgus x laius: 254x178 mm, 86 Illustrations, color; 9 Illustrations, black and white; IV, 146 p. 95 illus., 86 illus. in color. With online files/update., 1 Hardback
  • Sari: Methods in Molecular Biology 2963
  • Ilmumisaeg: 18-Sep-2025
  • Kirjastus: Springer-Verlag New York Inc.
  • ISBN-10: 1071647377
  • ISBN-13: 9781071647370
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Splice Switching: Methods and ProtocolsSuurem pilt
  • Formaat: Hardback, 146 pages, kõrgus x laius: 254x178 mm, 86 Illustrations, color; 9 Illustrations, black and white; IV, 146 p. 95 illus., 86 illus. in color. With online files/update., 1 Hardback
  • Sari: Methods in Molecular Biology 2963
  • Ilmumisaeg: 18-Sep-2025
  • Kirjastus: Springer-Verlag New York Inc.
  • ISBN-10: 1071647377
  • ISBN-13: 9781071647370
Teised raamatud teemal:
Püsilink: https://www.kriso.ee/db/9781071647370.html

This volume explores the latest advancements in the field of RNA therapeutics, with a particular focus on the development of exon skipping and inclusion therapies as targeted treatments for genetic diseases, as well as the evaluation methods used post-treatment. The chapters in this book cover both established methods and novel techniques used my researchers working on developing therapies that have proven to be promising for several diseases, including Duchenne muscular dystrophy (DMD), spinal muscular atrophy (SMA), and other genetic disorders characterized by abnormal splicing events. 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.

Cutting-edge and thorough, Splice Switching: Methods and Protocols is a valuable resource for researchers across various fields. This book will help in the advancement of basic RNA biology research and aid in accelerating therapeutic developments.

 

 DG9-Conjugated Morpholino-Based Exon 51 Skipping Therapy for Duchenne
Muscular Dystrophy.- DG9-Conjugated Morpholino Rescues Phenotype in Spinal
Muscular Atrophy Mice.- Cardio-Physiological Evaluations in A Dog Model with
Duchenne Muscular Dystrophy.- Splice Modulation Studies Using Urine-Derived
Cells.- Lipidomic Sample Preparation to Detect Phosphatidylcholine
Alterations in Skeletal Muscle.- Evaluation for the Efficacy of Exon Skipping
Therapy Using In Vivo Assessment of Skeletal Muscle Function and Fragility.-
Characterization and Phenotypic Assessment of Zebrafish Disease Models for
Splice Modulation Studies.- Preparation of Size-Defined PEG-Grafted
Copolymers as a Polymeric Nanoruler for Size Optimization in Passive
Targeting.- Splice Modulation Studies Using Reporter Mice.- Clinical Variant
Databases and Machine Learning Prediction Supporting Genomic Medicine.- In
Vivo Evaluation of CTG Repeat-Affected Muscle Pathology in a Myotonic
Dystrophy Model Mouse Using Electromyography and Fluorescence In Situ
Hybridization.- Utilization of Induced Pluripotent Stem Cell-Derived Neurons
to Investigate Splice-Modification Efficacy of Splice-Switching Drug
Candidates.- Development of Electric Field Stimulation (EFS)-Mediated
Skeletal Muscle Training with iPSCs.- Neurological Behavioral Assessment of
Exon Skipping Therapy for Neuromuscular Disorder Using Displaced Object
Recognition Test, Open Field Test, and Three-Chamber Social Approach Test.-
Analysis of Prefrontal-Amygdala Synaptic Functions Using Optogenetics and
Path-Clamp Recording.- Digital Outcome Measures for Gait and Spontaneous
Locomotor Activity in Canine Dystrophic Model.