Fig. 6: Safety analysis of RIDE in NHPs. | Nature Nanotechnology

Fig. 6: Safety analysis of RIDE in NHPs.

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

Fig. 6

a, Experimental workflow for the NHP study. The left putamen of NHPs was injected with PBS and the right putamen was injected with HTT-targeting RIDE. b, Brain MRI images taken before and after treatment. Pre-injection: repetition time (TR) = 2,100 ms, echo time (TE) = 3.3 ms, slice thickness = 1.0 mm, 104 slices. 21 days: TR = 6,000 ms, TE = 98 ms, slice thickness = 3.0 mm, 16 slices. c, Western blot analysis of HTT expression 21 days after RIDE injection. A representative result is shown from two biologically independent replicates. d–i, Analysis of immuno-related gene IL-6 (d), IL-1β (e), interferon-γ (IFN-γ) (f), IL-4 (g), IL-23 (h) and macrophage inflammatory protein-1α (MIP-1α) (i) expression by RT–qPCR. n = 9 NHP putamen samples collected 21 days after injection (three punches from each putamen) (n = 8 for RIDE group in g). j,k, Liver function tests. Sera were collected before injection and 21 days after injection. The serum ALT levels (j) and serum AST levels (k). n = 3 NHPs. Data and error bars represent mean ± s.e.m. Paired two-tailed Student’s t-tests.

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