Genome Editing in Neurosciences.

• Superior document: Research and Perspectives in Neurosciences Series
• Author: Jaenisch, Rudolf.
• TeilnehmendeR: Zhang, Feng.; Gage, Fred.
• Place / Publishing House: Cham : : Springer International Publishing AG,, 2017.; ©2017.
• Year of Publication: 2017
• Edition: 1st ed.
• Language: English
• Series: Research and Perspectives in Neurosciences Series
• Physical Description: 1 online resource (129 pages)

Table of Contents

• Intro
• Preface
• Contents
• List of Contributors
• In Vitro Modeling of Complex Neurological Diseases
• Introduction
• Induced Pluripotent Stem Cells to Model Complex Diseases
• Gene Editing to Generate Genetically Controlled Disease Models
• Functional Role of GWAS-Identified Risk Variants in Complex Disease
• Epigenomic Signatures to Prioritize GWAS-Identified Risk Variants
• Functional Analysis of Parkinsonś Disease-Associated Risk Variants
• Identification of Parkinsonś Disease-Associated Risk Variants in Brain-Specific Enhancer Elements
• Allele-Specific Gene Expression as a Robust Read-Out to Analyze Cis-Regulatory Effects
• Functional Analysis of Parkinsonś-Associated Risk Variants
• Mechanistic Study of Sporadic Diseases: Conclusions
• References
• Aquatic Model Organisms in Neurosciences: The Genome-Editing Revolution
• Introduction
• Zebrafish: With the CRiSPR-Cas9 System, Forward Genetic Screens Are Back Again
• Optimizing the Cripsr-Cas9 System in Transparent Marine Animals
• More and More Aquatic Model Organisms for Diversified Uses
• In Biomedical Research, Why and How Should We Use Aquatic Models to Study Diseases of the Nervous System?
• A Short Natural History of the Nervous System: Several Questions on Its Origin
• Conclusion
• References
• Genome-Wide Genetic Screening in the Mammalian CNS
• Introduction
• Genome-Wide Viral Library Preparation and Delivery
• Interpretation of Results
• Future Directions
• References
• CRISPR/Cas9-Mediated Knockin and Knockout in Zebrafish
• CRISPR/Cas9 and Gal4/UAS Combination for Cell-Specific Gene Inactivation
• Crispr/Cas9-Mediated Knockin Approaches in Zebrafish
• References
• Dissecting the Role of Synaptic Proteins with CRISPR
• Introduction
• Genome Editing Using CRISPR/Cas9
• Practical Considerations for the Use of CRISPR/Cas9.
• The Use of CRISPR/Cas9 in Neurons: Proof of Concept
• Conclusions and Future Perspectives
• References
• Recurrently Breaking Genes in Neural Progenitors: Potential Roles of DNA Breaks in Neuronal Function, Degeneration and Cancer
• References
• Neuroscience Research Using Non-human Primate Models and Genome Editing
• Introduction
• Characteristics of the Common Marmoset
• Advantages of Using Common Marmosets for Biomedical Research
• Transgenic Techniques and Genome Editing Technology for Marmoset Research
• Future Perspectives
• References
• Multiscale Genome Engineering: Genome-Wide Screens and Targeted Approaches
• Introduction
• Top-Down Approaches Using Genome-Wide CRISPR Screens
• Bottom-Up Approaches Using Exome Sequencing in Autism
• References
• Using Genome Engineering to Understand Huntingtonś Disease
• Huntington ́Disease
• Gene Editing Enzymes
• Uses for Gene Editing to Understand Human Diseases
• Gene Editing In Vivo to Treat Genetic Diseases
• Conclusion
• References
• Therapeutic Gene Editing in Muscles and Muscle Stem Cells
• Duchenne Muscular Dystrophy
• Current Gene-Targeted Therapeutic Strategies for DMD
• Challenges for Therapeutic Exon Skipping and Microdystrophin Delivery Strategies
• Gene-Editing Approaches to Restore Dystrophin Function in DMD
• Remaining Challenges for Therapeutic Development of DMD-CRISPR
• Challenges of DMD-CRISPR Delivery
• Potential Immune Response to Restored Dystrophin Protein
• Pre-existing and Acquired Immunity to Cas9
• Assessing Mutagenic Events at On-Target and Off-Target Sites
• Enabling HR for Precise Repair of Dmd
• Gene-Editing Therapy in Combination with AONs or Microdystrophin
• Possible Application of CRISPR-mediated gene editing Strategies in Other Diseases
• Conclusions and Perspective
• References.