Gene Therapy Models of Alzheimer’s Disease and Other Dementias
Fig. 1 rAAV2/5 efficiently transduces neurons and produces equal protein expression in the young and aged rat hippocampus (HP). (a–d) Young adult (a and c, 6 months, n = 3) and old…
NEUROLOGY
Viral Vector-Based Modeling of Neurodegenerative Disorders: Parkinson’s Disease
Model Construct Validity? Reproducible? Progressive nigrostriatal degeneration? Parkinsonian pathology? Parkinsonian symptoms? References Construct validity for idiopathic PD Within group Across groups Striatal DA terminal dysfunction/loss (over weeks) Terminal loss precedes…
Nonviral Gene Therapy of the Nervous System: Electroporation
Gene knockout Transgenic mice Viral system In vivo electroporation Toxicity No No Potential No Immunogenicity No No Potential No Time specificity No No Yes Yes Site specificity No No…
Ex Vivo Gene Therapy Using Human Mesenchymal Stem Cells to Deliver Growth Factors in the Skeletal Muscle of a Familial ALS Rat Model
Fig. 1 Ex vivo delivery of growth factor s targeting skeletal muscles for ALS . A GFP-expressing hMSC line was genetically modified by lentiviral infection to stably release growth factors…
MRI-Guided Delivery of Viral Vectors
Fig. 1 Infusion system setup: This schematic representation demonstrates all of the components in the infusion system. Starting with a 5 mL syringe (1) mounted onto an infusion pump (2),…
Gene Therapy of the Peripheral Nervous System: Celiac Ganglia
Fig. 1 Photograph of the celiac ganglia under a dissecting microscope. The celiac ganglia (circled) can be located by using the aorta, celiac artery, and superior mesenteric artery as landmarks…
Widespread Neuronal Transduction of the Rodent CNS via Neonatal Viral Injection
Fig. 1 Setup for free-hand intracerebroventricular injections. (a) Brace arms against the ice bucket for support and stability. Comfortably hold the syringe in one hand with the thumb positioned above…
Design of shRNA and miRNA for Delivery to the CNS
Fig. 1 An example of a microRNA designed against the human housekeeping gene GAPDH NM_002046.5 (NCBI), both flanks and loop have been modified and adapted from human miR155. (a) Indicates…
Altering Entry Site Preference of Lentiviral Vectors into Neuronal Cells by Pseudotyping with Envelope Glycoproteins
Fig. 1 Entry site preference of pseudotyped lentiviral vectors into neuronal cells. Lentiviral vector pseudotyped with VSV-G mainly enters the cell body areas of neurons, whereas the HiRet/NeuRet vectors with…
Altering Tropism of rAAV by Directed Evolution
Fig. 1 Library enrichment for target-specific variants. The DNA encoding the initial capsid library (top left) is cloned into the vector and enters the cycle through which target-specific variants are…
