Gene Therapy in Neurological Disorders covers viral and non-viral gene deliveries and tools, including emerging new technologies such as CRISPR/Cas9 gene editing. These tools can be applied to a range of disorders including neurodegeneration, neuroregeneration, muscular dystrophy, behavior disorders to retinal diseases. These successful examples will help scientists, physicians and students understand the principles of gene therapy and encourage them to use these tools in their research and translate the research findings into the clinic. The book begins with a detailed introduction about gene therapy, including the history of gene therapy, lessons learned, improvement, current status, prospects about gene therapies and their application in neurological disorders. Each chapter will give an in-depth introduction of the relevant field before diving into the specific tool or application. Covers experimental details of gene therapy tools applied for neuroscience research and neurological disordersWritten for researchers and clinical practitioners focusing on translational neuroscience, neurology, and gene therapiesDiscusses applications of gene therapy tools ranging from neurodegeneration, neuroregeneration, muscular dystrophy, behavior disorders to retinal diseases INDICE: 1. Opposing effects of viral mediated brain expression of apolipoprotein E2 (apoE2) and apoE4 on apoE lipidation and AB metabolism in apoE4-targeted replacement mice2. Potential role of orexin and sleep modulation in the pathogenesis of Alzheimer's disease3. Genetic Correction of Tauopathy Phenotypes in Neurons Derived from Human Induced Pluripotent Stem Cells4. Enhancing Astrocytic Lysosome Biogenesis Facilitates A Clearance and Attenuates Amyloid Plaque Pathogenesis5. Gene Therapy for the Treatment of Aromatic L-Amino Acid Decarboxylase (AADC) Deficiency6. Fiber-Modified Adenovirus for Central Nervous System Parkinson's Disease Gene Therapy7. Pharmacologically controlled, discontinuous GDNF gene therapy restores motor function in a rat model of Parkinson's disease8. Regulator of G-Protein Signaling-10 Negatively Regulates NF-B in Microglia and Neuroprotects Dopaminergic Neurons in Hemiparkinsonian Rats9. Striatal transplantation of human dopaminergic neurons differentiated from induced pluripotent stem cells derived from umbilical cord blood using lentiviral reprogramming10. In Vivo Evaluation of Candidate Allele-specific Mutant Huntingtin Gene Silencing Antisense Oligonucleotides11. Widespread and Efficient Transduction of Spinal Cord and Brain Following Neonatal AAV Injection and Potential Disease Modifying Effect in ALS Mice12. Therapeutic AAV9-mediated Suppression of Mutant SOD1 Slows Disease Progression and Extends Survival in Models of Inherited ALS13. Full-Length Dystrophin Reconstitution with Adeno-Associated Viral Vectors14. Correction of the Dystrophin Gene By the CRISPR/Cas9 System in a Mouse Model of Muscular Dystrophy15. Gene therapy for lysosomal storage diseases16. Prohibitin Viral Gene Transfer Protects Hippocampal CA1 Neurons From Ischemia and Ameliorates Postischemic Hippocampal Dysfunction17. Adenosine kinase, glutamine synthetase and EAAT2 as gene therapy targets for temporal lobe epilepsy18. Dual Transgene Expression in Murine Cerebellar Purkinje Neurons by Viral Transduction In Vivo19. Viral Transduction of Primary Schwann Cells Using a Cre-Lox System to Regulate GDNFExpression20. Transduction efficiency of neurons and glial cells by AAV-1,-5, -9, -rh10 and -hu11 serotypes in rat spinal cord following contusion injury
- ISBN: 978-0-12-809813-4
- Editorial: Academic Press
- Encuadernacion: Cartoné
- Páginas: 400
- Fecha Publicación: 01/05/2018
- Nº Volúmenes: 1
- Idioma: Inglés