Small molecule compounds with reconfirmed activity in hE72-Luc assay<sup>[a]</sup>, their known functions, cell cycle stage of action, activity and potency in hE72-Luc, HCI mitotic index and Alamar blue toxicity assays in HEK cells, as well as exon skipping activity on the hE72-Luc construct in HEK
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[a] Compound classed as reconfirmed hit when luciferase activity of purified powder was >2 fold above DMSO in duplicate plates; [b] FC = fold change above DMSO; [c] assay was performed in the presence of 0.5 µM AON and 1 µM compound in HEK cells transfected with hE72-Luc and data represents average fold change in the normalized skip/full-length ratio; [d] assay was performed in the absence of AON and presence of 1 µM compound in mdx-H2K cells and data represents average fold change in the normalized skip/full-length ratio; [e] assay was performed in the presence of 0.2 µM AON and presence of 1 µM compound in mdx-H2K cells and data represents average fold change in the normalized skip/full-length ratio; [f] not determined; [g] not applicable.
