Extended Data Fig. 2: ASOs efficiently penetrate into and regulate gene expression in 3D human organoids in culture.
From: Therapeutically viable generation of neurons with antisense oligonucleotide suppression of PTB
a, Representative immunofluorescence images from a 5-month-old human organoid taken 1-week post Cy3-Malat1-ASO addition to the culture medium. Images show a view of (red) Cy3-Malat1-ASO (visualized by direct immunofluorescence) or (blue) DAPI staining for DNA; experiment was reproduced two times, independently, with similar results. b, Malat1 mRNA levels measured by qPCR 2 or 4 weeks after addition to human organoid cultures or either 10 μM Malat1-ASO or control, non-targeting ASO. Data are presented as fold change mean +/− s.e.m. (2 weeks control mean: 1 +/− 0.04; 2 weeks post Malat1-ASOs mean: 0.11 +/− 0.03; 4 weeks control mean: 1 +/− 0.1; 4 weeks post Malat1-ASOs mean: 0.1 +/− 0.02; n = 3 biological repeats). c, Representative immunofluorescence images from 5-month-old human organoids taken 1-month post-Malat1-ASO application to the organoid culture medium. Images show a view of (green) a Malat1-ASO (visualized by immunofluorescence) or (blue) DAPI staining for DNA; experiment was reproduced three times, independently, with similar results.
