Viral vectors for gene therapy methods and protocols - Manfredsson, Fredric P.; Benskey, Matthew J. (Springer New York, 2019)
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