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…
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…
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…
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…
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…
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°…
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…
Serotype Natural host/origin Optimal organ/tissue/cell type transduced CNS area transduced Receptor Coreceptor Blood–brain barrier penetration Disease targets AAV1 Monkey Muscle [39], Liver [110], CNS [32, 42] SNpc, SNpr, midbrain, hippocampus…
Fig. 1 (a) Nine copies of Epo HRE consensus sequence. The HIF-1 binding sites are underlined. (b) Schematic drawing of H9LacZ and H9VEGF. Nine copies of Epo HRE (9HRE) were…
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)…
