Gene Therapy for Neurological Disorders

Fredric P. Manfredsson, Ph.D. Assistant Professor Michigan State University Division of Translational Science & Molecular Medicine Van Andel Institute 333 Bostwick Ave NE Grand Rapids, MI 49503 Tel: 616-234-0968 Fax: 616-234-0990

Introduction to Viral Vectors and Other Delivery Methods for Gene Therapy of the Nervous System.- Delivering Transgenic DNA Exceeding the Carrying Capacity of AAV Vectors.- Expression of Multiple Functional RNAs or Proteins from One Viral Vector.- Regulated Gene Therapy.- Design of shRNA and miRNA for Delivery to the CNS.- Tissue Specific Promoters in the CNS.- Small Scale Recombinant Adeno-Associated Virus Purification.- Lentivirus Production and Purification.- Viral Vector Production: Adenovirus.- Controlling AAV Tropism in the Nervous System with Natural and Engineered Capsids.- Altering Tropism of rAAV by Directed Evolution.- Altering Entry Site Preference of Lentiviral Vectors into Neuronal Cells by Pseudotyping with Envelope Glycoproteins.- Directed Evolution of Adenoviruses.- Intraparenchymal Stereotaxic Delivery of rAAV and Special Considerations in Vector Handling.- MRI-Guided Delivery of Viral Vectors.- Systemic Gene Therapy for Targeting the CNS.- Widespread Neuronal Transduction of the Rodent CNS via Neonatal Viral Injection.- AAV-Mediated Gene Transfer to Dorsal Root Ganglion.- Gene Therapy of the Peripheral Nervous System: The Enteric Nervous System.- Gene Therapy of the Peripheral Nervous System: Celiac Ganglia.- Convection Enhanced Delivery of Recombinant Adeno-Associated Virus into the Mouse Brain.- Non-Viral Gene Therapy of the Nervous System: Electroporation.- Non-Viral, Lipid-Mediated DNA and mRNA Gene Therapy of the Central Nervous System (CNS): Chemical-Based Transfection.- Ex Vivo Gene Therapy Using Human Mesenchymal Stem Cells to Deliver Growth Factors in the Skeletal Muscle of a Familial ALS Rat Model.- Gene Therapy Models of Alzheimer’s Disease and Other Dementias.- Viral Vector-Based Modeling of Neurodegenerative Disorders: Parkinson’s Disease.- Gene Therapy-Based Modeling of Neurodegenerative Disorders: Huntington's Disease.- Gene Therapy for the Treatment of Neurological Disorders: AmyotrophicLateral Sclerosis.- Stereotaxic Surgical Targeting of the Non-Human Primate Causate and Putamen: Gene Therapy for Huntington’s Disease.- Gene-Therapy for the Treatment of Neurological Disorders: Metabolic Disorders.- Gene Therapy for the Treatment of Neurological Disorders: Central Nervous System Neoplasms.- AAV2-Neurturin for Parkinson’s Disease: What Lessons Have We Learned?.