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. 2019 Apr 25;16:686–697. doi: 10.1016/j.omtn.2019.04.018

Figure 2.

Figure 2

Screening of NPC1-Specific LNA-ASOs in Human and Murine Cell Lines

(A and B) Human HeLa and THP1 cells (A) as well as murine cell line 4T1 (B) were treated with 10 μM of ASOs. After 3 days cell lysates were used to determine NPC1 and HPRT1 mRNA levels. (A) Shown is the correlation of residual NPC1 mRNA expression (means and SD of triplicate wells) after treatment in HeLa (x axis) versus THP1 cells (y axis). Values were normalized to HPRT1 and relative to the untreated control (set as 1; empty square). ASO 05HM is indicated as a filled diamond, ASO 28H as a filled triangle, Neg1 as an empty circle, and other NPC1-specific ASOs as filled circles. (B) Residual Npc1 mRNA expression in 4T1 cells after treatment with respective NPC1-specific ASOs or negative control Neg1. Values were normalized to Hprt1 and are shown relative to the untreated control (set as 1). Error bars show SD (triplicate wells). The number of mismatches to murine Npc1 sequence of human specific ASOs (H) are shown in different colors and patterns.