Gene silencing with virally delivered shRNA represents a promising approach for treatment of inherited neurodegenerative disorders. In the present study we develop a subpial technique, which we show in adult animals successfully delivers adeno-associated
We evaluated the quantity of viral DNA from the serotypes AAV9, AAV6, and a mosaic AAV1&2, expressing green fluorescent protein (GFP) under the neuron-specific synapsin promoter (syn). AAVs were administered intravenously during MRIgFUS targeting to the striatum and hippocampus in mice. The...
49 (B) Schematic illustration of the differentiation process from Hb9:GFP ESCs to spinal MNs and INs. RA, retinoic acid; SAG, Smoothened agonist; ESC, embryonic stem cell; MN, motor neuron; IN, interneuron. (C and D) Heatmaps (transcripts per million [TPM]) presenting miRNA abundance. ...
Focused ultrasound (FUS) delivery of AAV6-syn-GFP did not result in a statistically significant increase in percentage of GFP-positive neurons compared to AAV1&2-syn-GFP in the total GFP-positive region of the brain (p = 0.08) (a) and hippocampus (p = 0.09) (b). There was...
To refine this approach, the present study characterizes the transduction profiles of new self-complementary AAV9 (scAAV9) expressing the green fluorescent protein (GFP) either under an astrocyte (glial fibrillary acidic (GFA) protein) or neuronal (Synapsin (Syn)) promoter, after intravenous ...
To refine this approach, the present study characterizes the transduction profiles of new self-complementary AAV9 (scAAV9) expressing the green fluorescent protein (GFP) either under an astrocyte (glial fibrillary acidic (GFA) protein) or neuronal (Synapsin (Syn)) promoter, after intravenous ...