Genome Editing in Neurosciences. - Jaenisch, Rudolf; Zhang, Feng.; Gage, Fred. (Cham : Springer International Publishing AG, 2017)
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.
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Electronic reproduction. Ann Arbor, Michigan : ProQuest Ebook Central, 2023. Available via World Wide Web. Access may be limited to ProQuest Ebook Central affiliated libraries.
Keywords: Electronic books