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The second edition of Translational Neuroscience offers wide-ranging and insightful perspectives on the effort to bring potentially revolutionary new classes of therapies to the clinic, thereby transforming the treatment of human nervous system disorders. Great advances in the fields of basic neuroscience, molecular biology, genomics, gene therapy, cell therapy, stem cell biology, information technology, rehabilitation, and others over the last 20 years have generated unprecedented opportunities to treat heretofore untreatable disorders of the nervous system. This book provides detailed coverage of these efforts, together with the methods for pursuing clinical translation and assessing clinical outcomes. All of the first edition chapters have been extensively updated, and many new chapters have been added, including brain-machine interface, gene therapy, anti-sense oligonucleotides, and CRISPR. Among the many other topics covered are Alzheimer's disease, Parkinson's disease, stroke, multiple sclerosis, epilepsy, motor neuron disease, pain, inborn errors of metabolism, brain tumors, spinal cord injury, neuroprosthetics, rehabilitation, and clinical trial design/consideration.
Mark H. Tuszynski, MD, PhD, is Distinguished Professor of Neurosciences and Director of the Translational Neuroscience Institute at the University of California - San Diego, and a neurologist at the Veterans Administration Medical Center in La Jolla, California.
I Introduction II Molecular Therapies.- Gene Therapy, Anti-Sense Oligonucleotides, RNA Interference, and CRISPR.- Gene Therapy Tools for Translational Neuroscience.- AAV9 SMN1 Gene Therapy for Spinal Muscular Atrophy.- Gene Therapy for Retinitis Pigmentosa.- NGF and BDNF Gene Therapy for Alzheimer's Disease.- Proganulin Gene Therapy in Alzheimer's Disease.- AADC Gene Therapy for Congenital Dopamine Deficiency.- Gene Therapy for Inborn Errors of Metabolism.- GDNF Gene Therapy forParkinson's Disease.- GDNF Gene Therapy for Parkinson's Disease.- Gene Therapy for Spinal Cord Injury.- GAD Gene Therapy for Epilepsy.- Gene Therapy for Pain.- Anti-Sense Oligonucleotide Gene Therapy for Huntington's Disease.- Anti-Sense Oligonucleotide Gene Therapy for Spinocerebellar Ataxia.- Anti-Sense Oligonucleotides for Amyotrophic Lateral Sclerosis.- Micro-RNA Gene Therapy for Huntington's Disease.- Targeting C9orf72 Mutations in Frontotemporal Dementia.- CRISPR-Cas9 Therapy for Alzheimer's Disease III Stem Cells.- Stem Cells for Amyotrophic Lateral Sclerosis.- iPSCs for Parkinson's disease.- Stem Cells for Huntington's disease.- Stem Cells for Multiple Sclerosis.- Stem Cells for Inborn Errors of Metabolism.- Stem Cells for Spinal Cord Injury.- Induced Pluripotent Stem Cells for Retinal Disease.- Bone Marrow Stromal Cells for Stroke.- Glioma Stem Cells.- IV Pharmacological Approaches to Neural Therapy.- Amyloid-Modulating Therapies for Alzheimers Disease.- Novel Pharmacological Therapies for Alzheimer's Disease.- Down's Syndrome.- Spinal Cord Injury.- Nogo Inhibition in SCI.- Intrinsic Neuronal Mechanisms in Spinal Cord Injury.- Translational Neuroscience and Pain.- Neurodevelopmental Disorders and Autism.- V Activity Dependent Plasticity and Neurorehabilitation.- Epidural Spinal Stimulation for Spinal Cord Injury.- Neural Prostheses for Neurotrauma.- Neuroengineering in Rehabilitation.- Deep Brain Stimulation for Neuropsychiatric Disorders.- Brain Machine Interface for Spinal Cord Injury.- Pharmacological Approaches to Enhance Stroke Recovery.- VI Conclusion.
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