Genome Editing for Neurodegenerative Diseases: From Concept to Clinical Trials examines recent advances in neurodegenerative disease research and clinical challenges in practical applications. With an emphasis on genome editing tools such as ZFNs, TALENSs and CRISPR-Cas, this book sheds light on the possibilities to counteract genetic and neurodegenerative diseases. It will be of interest to researchers in neuroscience and biopharmaceuticals, as well as clinicians specializing in neurodegenerative diseases.
1. Introduction - p. xiii 2. Chapter one. Therapeutic potential of genome editing tools in neurodegenerative diseases - p. 1 3. Chapter two. Genetic and molecular basis of neurodegenerative diseases - p. 19 4. Chapter three. Genome editing tools: principles, structures, and mechanisms - p. 47 5. Chapter four. Advances in CRISPER/Cas system and genome editing technologies for the treatment of neurodegenerative diseases - p. 69 6. Chapter five. Use of CRISPR system in genetic screening to detect neurodegenerative disease - p. 91 7. Chapter six. Epigenetic manipulation and gene regulation using the CRISPR/Cas system for neurodegenerative diseases - p. 119 8. Chapter seven. Transgene delivery system: viral, nonviral, and other methods for central nervous system - p. 135 9. Chapter eight. Gene therapies for neurodegenerative disease: current knowledge and challenges - p. 157 10. Chapter nine. Gene therapy under clinical trials for neurodegenerative diseases - p. 177 11. Chapter ten. Clinical challenges and future prospective in use of gene-editing tools as neurotherapeutics - p. 197 12. Index - p. 227
Genome Editing for Neurodegenerative Diseases: From Concept to Clinical Trials examines recent advances in neurodegenerative disease research and clinical challenges in practical applications. With an emphasis on genome editing tools such as ZFNs, TALENSs and CRISPR-Cas, this book sheds light on the possibilities to counteract genetic and neurodegenerative diseases. It will be of interest to researchers in neuroscience and biopharmaceuticals, as well as clinicians specializing in neurodegenerative diseases.
1. Introduction - p. xiii 2. Chapter one. Therapeutic potential of genome editing tools in neurodegenerative diseases - p. 1 3. Chapter two. Genetic and molecular basis of neurodegenerative diseases - p. 19 4. Chapter three. Genome editing tools: principles, structures, and mechanisms - p. 47 5. Chapter four. Advances in CRISPER/Cas system and genome editing technologies for the treatment of neurodegenerative diseases - p. 69 6. Chapter five. Use of CRISPR system in genetic screening to detect neurodegenerative disease - p. 91 7. Chapter six. Epigenetic manipulation and gene regulation using the CRISPR/Cas system for neurodegenerative diseases - p. 119 8. Chapter seven. Transgene delivery system: viral, nonviral, and other methods for central nervous system - p. 135 9. Chapter eight. Gene therapies for neurodegenerative disease: current knowledge and challenges - p. 157 10. Chapter nine. Gene therapy under clinical trials for neurodegenerative diseases - p. 177 11. Chapter ten. Clinical challenges and future prospective in use of gene-editing tools as neurotherapeutics - p. 197 12. Index - p. 227
