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. …
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…
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…
Fig. 1 Dosing study showing transduction in coronal sections of healthy rat striatum following infusion of three different concentrations of AAV5-CAG-GDNF. Titer of the undiluted vector was 2 × 1013 GC/ml. The…
Fig. 1 Schematic of the exon 7 regulation within SMN pre-mRNA and trans-splicing strategy to replace the SMN2 exon 7. (a) SMN pre-mRNA is regulated by cis elements (within intron…
