Genome Editing in Neurosciences.
| Main Author: | |
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| Other Authors: | , |
| Format: | eBook |
| Language: | English |
| Published: |
Cham :
Springer International Publishing AG,
2017.
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| Edition: | 1st ed. |
| Series: | Research and Perspectives in Neurosciences Series
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| Subjects: | |
| Online Access: | Click to View |
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.