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…
NEUROLOGY
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)…
Gene Delivery and Gene Therapy for Alzheimer’s Disease
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…
Targeted Gene Therapy for Ischemic Stroke
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…
Gene Therapy Approaches Using Reproducible and Fully Penetrant Lentivirus-Mediated Endogenous Glioma Models
Author (reference) Animal System Age/injection site Proliferation (RTK/KRAS/PI3K) Apoptosis (p53/ARF) Cell cycle (Rb/INK4) Best combined penetrance Holland et al. 2000 [13] Mice RCAS Neonatal/R frontal AKT, KRAS 26 % Dai…
Gene Therapy Approaches to Promoting Axonal Regeneration After Spinal Cord Injury
Fig. 1 Behavioral tests for the functional recovery after rubrospinal tract transection. (a) Photo showing the symmetrical use of both forelimbs of the rat during vertical exploration of the cylinder…
Disorders of the Cerebrovascular System
Stroke type Percentage (% of each category) Ischemic 85 Carotid artery circulation 55 Vertebral/basilar artery circulation 12 Lacune 31 Other (vasculopathy, coagulopathy, sickle cell, hyperviscosity, vasculitis) 2 Hemorrhagic 14 Hypertensive…
