Figure 4.
In vitro assays to assess compound binding to tau RNA. (A) 2-AP assay. Addition of compound quenches emission. No data are available for 9 due to spectral overlap with 2-AP. (B) Fluorescence resonance energy transfer (FRET) FRET-based quenching (Q-)assay. (C) FRET-based U1 snRNA assay. To the right of the schemes are plots showing correlation between each in vitro binding assay and exon 10 skipping determined at 10 μM for all compounds except 6 and 7, which were determined at 12.5 μM. Exon skipping assays were carried out with HeLa cells transfected with a DDPAC mini-gene. Error bars indicate SD.