Genome Editing for Neurodegenerative Diseases: From Concept to Clinical Trials

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. Covers basic mechanisms of genome-editing tools and their therapeutic potential Highlights current research progress and the therapeutic strategies to use genome-editing tools against neurodegenerative diseases Includes research on genome-editing tool development from the basic concept to clinical trials INDICE: 1. Overview of genome editing tools and general neurodegenerative diseases 2. Genetic and molecular basis of neurodegenerative diseases 3. Genome editing tools: Principles, structures, and mechanisms 4. Role of genome editing tools in the establishment of neurodegenerative disease models: In-vitro and In-vivo studies 5. Innovation of CRISPR/Cas systems and genome editing approaches for neurodegenerative diseases 6. Use of CRISPR systems in genetic screening to investigate neurodegenerative diseases 7. Epigenetic manipulation and genes regulation using CRISPR/Cas system for neurodegenerative diseases 8. Transgene delivery systems: Viral, non-viral and other methods for CNS 9. Gene-therapies for neurodegenerative diseases: current knowledge and challenges 10. Gene therapies under clinical trials for neurodegenerative diseases 11. Clinical challenges and future prospective in use of gene-editing tools as neurotherapeutics

• ISBN: 978-0-443-23826-0
• Editorial: Academic Press
• Encuadernacion: Rústica
• Páginas: 400
• Fecha Publicación: 01/12/2024
• Nº Volúmenes: 1
• Idioma: Inglés