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.