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 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…
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 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…
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…
Lentiviral Vectors for Gene Delivery to the Nervous System
Pseudotype envelope Tropism and other features References Amphotropic MLV Efficient transduction of hematopoietic progenitor cells, T lymphocytes Reviewed in [60] Aura virus Transduction of monocyte derived dendritic cells (DCs), useful…
Gene Delivery to Neurons of the Dorsal Root Ganglia Using Adeno-Associated Viral Vectors
Fig. 1 Configuration of the stereotactic frame for injection of rat dorsal root ganglia. (a) First the electrode holder is placed in inverted orientation. (b) The manipulator is swiveled 180°…
Gene Therapy for Epilepsies
Target gene Injection region Vector Experimental model Functional outcome References GAD Substantia nigra Engineered cells overexpressing GAD65 Kindling Increased seizure threshold Thompson et al. [66] Substantia nigra Engineered cells overexpressing…
Gene Therapy for Huntington’s Disease
Volume Reagent Final concentration Complete DMEM media 500 mL DMEM (Invitrogen #12430-062) 50 mL Fetal bovine serum (Thermo HyClone #SH30406.02) 9 % 5 mL Non-essential amino acids (100×, Invitrogen, #11140050)…
