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.

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.

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.