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Journal: Molecular Therapy Advances
Article Title: Single-cell and spatial transcriptomic analyses of gene therapy-associated retinal inflammation in non-human primates
doi: 10.1016/j.omta.2026.201726
Figure Lengend Snippet: Robust expression of human RPE65 and RPGR transgenes in AAV-treated NHP retinas (A and D) Immunostaining of retina sections from AAV2-CAG- hRPE65 (NHP1) and AAV8-GRK1- hRPGRco (NHP2) treated areas detect both native NHP and transgene-derived human RPE65 and RPGR proteins, respectively. Control sections were taken from outside the treated blebs. GCL, ganglion cell layer; INL, inner nuclear layer; OPL, outer plexiform layer; ONL, outer nuclear layer; PL, photoreceptor layer; RPE, retinal pigmented epithelium; CH, choroid. Scale bars, 20 μm. (B and E) Spatial transcriptomic maps of native versus human RPE65 (NHP1) and RPGR (NHP2) gene expression within retina sections. Red spots represent the locations of cell clusters expressing the gene of interest overlayed on the H&E-staining image. Scale bars, 0.5 mm. (C and F) scRNA-seq reveals the levels of transgene expression by each retinal cell type. Circle size represents the proportion of cells that express the transgene within each population, while color intensity represents the mean expression level per cell. AC, amacrine cells; BPC, bipolar cells; HC, horizontal cells; MG, Müller glia.
Article Snippet:
Techniques: Expressing, Immunostaining, Derivative Assay, Control, Gene Expression, Staining
Journal: Molecular Therapy Advances
Article Title: Single-cell and spatial transcriptomic analyses of gene therapy-associated retinal inflammation in non-human primates
doi: 10.1016/j.omta.2026.201726
Figure Lengend Snippet: Distribution of mononuclear phagocytes after AAV gene therapy in NHPs (A and C) Spatial transcriptomic mapping of monocytic phagocytes (including microglia) over treated versus untreated retinal sections from NHP1 (A) and NHP2 (C). Red spots represent cells expressing the marker genes P2RY12 and HEXB . Scale bars, 0.5 mm. (B and D) Immunostaining for IBA1 demonstrates amoeboid-shaped monocytic phagocytes, consistent with activation. Scale bars, 20 μm.
Article Snippet:
Techniques: Expressing, Marker, Immunostaining, Activation Assay