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…
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…
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…
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),…
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…
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…
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…
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…
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…
Fig. 1 Intraganglionic injection. Paravertebral surgical exposure for intraganglionic injection, illustrating the operative field (left panel), reference diagram (middle panel), and cleaned vertebral bones (right panel). (a) Initial dissection of…
