Regulated Gene Therapy
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…
Directed Evolution of Adenoviruses
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…
Introduction to Viral Vectors and Other Delivery Methods for Gene Therapy of the Nervous System
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. …
Lentivirus Production and Purification
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…
Adeno-Associated Viral Gene Therapy for Retinal Disorders
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…
AAV Gene Therapy Strategies for Lysosomal Storage Disorders with Central Nervous System Involvement
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…
Gene Therapy in Spinal Muscular Atrophy (SMA) Models Using Intracerebroventricular Injection into Neonatal Mice
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…
Gene Therapy for Parkinson’s Disease: AAV5-Mediated Delivery of Glial Cell Line-Derived Neurotrophic Factor (GDNF)
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…
Adeno-Associated Vectors for Gene Delivery to the Nervous System
Plasmid Ratio Amount per plate (μg) Amount per 30 dishes transfection (μg) Helper plasmid 2 26 780 Packaging plasmid 1 13 390 cis plasmid pCMV-GFP 1 13 390 Transfection by…
Gene Therapy for Chronic Pain: How to Manipulate and Unravel Pain Control Circuits from the Brain?
Fig. 1 Diagram depicting the organization of circuits involved in pain facilitation from the DRt. This medullary area is involved in a feedback reciprocal loop with the spinal cord, which…
