Gene Therapy in Neurological Disorders

Table of Contents

Section I  Overview and Methods 1. Gene therapy methods and their applications in neurological disorders 2. Targeting transgene and RNA interference-based gene silencing sequences to astrocytes using viral vector-mediated approaches 3. Gene therapy using genomic DNA: Advances and challenges 4. Stem cell-based gene therapy in neurological disorders

Section II  Approaches to specific disorders 5. Targeting astrocytes with viral gene therapy for Alzheimer's disease 6. Prophylactic and therapeutic applications of catalytic immunoglobulin gene delivery in a mouse model of Alzheimer's disease 7. Lentiviral vector delivery of orexin gene to study potential role of orexin and sleep modulation in the pathogenesis of Alzheimer's disease 8. Pharmacologically controlled neurotrophic factor gene therapy for Parkinson´s Disease 9. Gene therapy as a novel target for treatment of symptoms and medication complications in Parkinson's disease 10. Inhibition of aggregation of mutant huntingtin by nucleic acid aptamers in vitro and in a yeast model of Huntington's disease 11. Intraspinal delivery of recombinant AAV mediated Interleukin-10 modifies disease in ALS mice. 12. Differentiation of human induced pluripotent stem cells to Purkinje neurons 13. Expressing full-length dystrophin using adeno-associated virus 14. Genome editing therapy for Duchenne muscular dystrophy 15. AAV vector mediated gene delivery to neurons and glial cells in damaged spinal cord 16. Transplantation of human induced pluripotent stem cell-derived neural precursor cells for treatment of spinal cord injury 17. Viral transduction of primary Schwann cells using a Cre-lox system to regulate GDNF expression 18. Application of Herpes simplex virus vectors in treatment of neuropathic pain 19. Oncolytic virotherapy for gliomas: a preclinical and clinical summary

Authors

Mingjie Li Assistant Professor, Department of Neurology, Washington University School of Medicine, St. Louis, Missouri, USA. Dr. Li is an Associate Professor in the Department of Neurology at Washington University School of Medicine in St. Louis, Missouri. He is also a Senior Scientist in the Viral Vectors Core at the Hope Center for Neurological Disorders. Dr. Li has over 15 years' experience in viral vector development and gene therapy, and some of the vectors have been used in clinical trials. After joining Department of Neurology at Washington University, Dr. Li has focused on developing vectors for applications in neuroscience and constructed numerous new vectors targeting neurological disorders. B. Joy Snider Associate Professor, Director of Hope Center Viral Vectors Core, Department of Neurology, Washington University School of Medicine, St. Louis, Missouri, USA. Dr. Snider is a Professor in the Department of Neurology at Washington University School of Medicine. She is also the Director of Hope Center Viral vectors Core. Dr. Snider is interested in translational research on neurodegenerative disorders and undertakes clinical studies at the Washington University Alzheimer's Disease Research Center. She also sees patients at Barnes-Jewish Hospital/Washington University in St. Louis, Missouri