Viral Vectors for Gene Therapy

Basic Concepts in Viral Vector-Mediated Gene Therapy.- Design and Assembly of CRISPR/Cas9 Lentiviral and rAAV Vectors for Targeted Genome Editing.- Design, Construction, and Application of Transcription Activation-Like Effectors.- Practical Considerations for the use of DREADD and Other Chemogenetic Receptors to Regulate Neuronal Activity in the Mammalian Brain.- AAV Production using Baculovirus Expression Vector System.- Multimodal Production of Adeno-Associated Virus.- Generation of High Titer Pseudotyped Lentiviral Particles.- A Scalable Lentiviral Vector Production and Purification Method using Mustang Q Chromatography and Tangential Flow Filtration.- Current Use of Adenovirus Vectors and their Purification Methods.- Construction of Oncolytic Herpes Simplex Virus with Therapeutic Genes of Interest.- Poxviruses as Gene Therapy Vectors: Generating Poxviral Vectors Expressing Therapeutic Transgenes.- AAV Mediated Gene Delivery to the Mouse Liver.- Surgical Methods for Inner Ear Gene Delivery in Neonatal Mouse.- Gene Transfer to Mouse Kidney In Vivo.- Co-Delivery of a Short-Hairpin RNA and a shRNA-Resistant Replacement Gene with Adeno-Associated Virus: An Allele-Independent Strategy for Autosomal Dominant Retinal Disorders.- Localized Intra-Arterial Gene Delivery using AAV.- Stable Genetic Modification of Mesenchymal Stromal Cells Using Lentiviral Vectors.- Systemic Delivery of Adeno-Associated Viral Vectors in Mice and Dogs.- Intrathecal Delivery of AAV Vectors in Cynomolgus Macaques for CNS Gene Therapy and Gene Expression Analysis in Microdissected Motor Neurons.- Detailed Method for Intrathecal Delivery of Gene Therapeutics by Direct Lumbar Puncture in Mice.- Cerebromedullary Cistern Injection of Viral Vectors in Non-Human Primates.