© 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…
Fig. 1 The viral genomic sequence immediately surrounding the F421Y mutation (bold) is indicated. Note that the pol gene is on the complementary strand (translated) as the adenovirus genome 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. …
Fig. 1 GFP expression following transduction with a VSV-G pseudotyped LV vector. LV vectors produced using the current protocol efficiently transduces cells in vitro (a–c) and in vivo (d). SH-SY5Y…
Fig. 1 Basic vertebrate retinal structure image showing the various layers of the vertebrate retina with light passing through the ganglion cell layer (GCL), inner nuclear layer (INL) to the…
Fig. 1 Lysosomal enzymes involved in the catabolism of GM1 ganglioside to GM3 ganglioside and the lysosomal storage disorders associated with loss of each enzyme Adeno-associated virus is a replication-deficient…
