Extended Data Fig. 4: Pseudotyping RIDE with hyRV-G for neuron-specific depletion of Htt in vivo. | Nature Nanotechnology

Extended Data Fig. 4: Pseudotyping RIDE with hyRV-G for neuron-specific depletion of Htt in vivo.

From: Customizable virus-like particles deliver CRISPR–Cas9 ribonucleoprotein for effective ocular neovascular and Huntington’s disease gene therapy

Extended Data Fig. 4

a, Experimental workflow and schematic diagram of stereotaxic injection. RIDE-Htt (21.5 ng p24) was injected into the right striatum of adult C57BL6/J mice. After 1 or 2 weeks, the mice were euthanized. b, Deep-sequencing analysis of the on-target effects in the right striatum (2 mm lateral and 0.5 mm rostral to the bregma at 3 mm depth). n = 4 mice. Control versus RIDE, *P = 0.0271. c, Western blot analysis of HTT expression in the brain. The right striatum of mice was injected with Htt-targeting RIDE or IDLV-GFP. The number below each lane indicates the relative expression level of HTT. A representative result is shown from three biologically independent replicates. d, Confocal microscopy analysis of potential neuron injury induced by RIDE injection. Scale bar, 50 μm. A representative image from four mice is shown. Data and error bars represent the mean ± SEM. Unpaired two-tailed Student’s t-tests for b.

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