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. 2013 Mar 19;2(3):e81. doi: 10.1038/mtna.2013.9

Figure 5.

Figure 5

(CUG)n length selectivity in DM1 myoblasts. (a) Human myoblasts expressing either hDMPK (CUG)21 and (CUG)200 transcripts (21/200) or hDMPK (CUG)5 and (CUG)1400 transcripts (5/1400) were treated with a selection of CAG AONs. Northern blot analysis indicated that expanded hDMPK transcripts (“exp”, arrow heads) were efficiently silenced by all AONs. (CUG)21 transcripts (“normal”, double arrow heads) were moderately silenced. Only RNase-H recruiting PS260 substantially degraded (CUG)5 transcripts (“normal”, double arrow head). One representative blot of three independent experiments is shown for each cell culture. Quantification of signals is summarized in the graphs at the bottom. *P < 0.05; **P < 0.01; ***P < 0.001. (b) A significant correlation was demonstrated between the number of CTG triplets and silencing activity of PS147, PS58, and PS146 (P < 0.05, Pearson's correlation, r = −0.98), but not PS260. AON, antisense oligonucleotide; GAPDH, glyceraldehyde 3-phosphate dehydrogenase; hDMPK, human DM protein kinase.