Route of administration Advantages Disadvantages Intramuscular (IM) Noninvasive Requires retrograde transport of the vector; impractical to treat all skeletal muscles Intraparenchymal (IP) Delivery of therapeutic directly to location of interest…
© Springer Science+Business Media New York 2016Fredric P. Manfredsson (ed.)Gene Therapy for Neurological DisordersMethods in Molecular Biology138210.1007/978-1-4939-3271-9_27 27. Gene Therapy-Based Modeling of Neurodegenerative Disorders: Huntington’s Disease Deborah Young1 (1) Department of Pharmacology & Clinical Pharmacology…
Fig. 1 Representative images of mouse brain following systemic injection of AAV9 CB GFP. GFP immunofluorescence shows extensive GFP expression in neurons throughout multiple structures including the cortex (A), hippocampus…
Fig. 1 Transcriptional targeting of AAV vectors in the rat striatum. Exclusively neuronal transgene expression is shown after AAV-5-hSYN-EGFP transduction (left panel; shown is an overlay of green = EGFP fluorescence with…
© Springer Science+Business Media New York 2016Fredric P. Manfredsson (ed.)Gene Therapy for Neurological DisordersMethods in Molecular Biology138210.1007/978-1-4939-3271-9_23 23. Non-Viral, Lipid-Mediated DNA and mRNA Gene Therapy of the Central Nervous System (CNS): Chemical-Based Transfection James G. Hecker1 …
Optiprep 5 M NaCl 5×TD Water Final volume 15 % 45 ml 36 ml 36 ml 63 ml 180 ml 30 % 60 ml – 24 ml 36 ml…
Strategy Products Stoichiometry Advantages Disadvantages Potential applications References Fusion protein (Fig. 1a) Protein 1:1 predictable + Intracellular co-localization of the two proteins is guaranteed + Very space-efficient thus good for…
© Springer Science+Business Media New York 2016Fredric P. Manfredsson (ed.)Gene Therapy for Neurological DisordersMethods in Molecular Biology138210.1007/978-1-4939-3271-9_10 10. Controlling AAV Tropism in the Nervous System with Natural and Engineered Capsids Michael J. Castle1, 4, Heikki T. Turunen5, Luk H. Vandenberghe5…
Fig. 1 Schematic representation of the Tet-Off (left ) and Tet-On (right ) system, in the presence (higher panel ) or absence (lower panel ) of tetracycline (red squares). Tet-Off: the transactivator (tTA) is…
Vector Capacity Flexibility Considerations rAAV 4.7-5 kb Tropism can be changed by pseudotyping and directed evolution. The capsid may illicit an immune response when delivered peripherally, or with readministration centrally. …
