Table 1 Potencies and selectivities of compounds 1–4 *.

From: Induction and reversal of myotonic dystrophy type 1 pre-mRNA splicing defects by small molecules

Potencies and selectivities of small molecules

Compound

IC50† (μM)

Complex

IC50 of 1; selectivity for r(CUG)exp–MBNL1‡

IC50 of 2; selectivity for r(CUG)exp–MBNL1§

1

52±12

r(CUG)12–MBNL1

54±2; -

2±0.4; -

2

2±0.4

r(CAG)12–MBNL1

66±13; 1.2

37±5; 20

3

242±1

r(CGG)12–MBNL1

54±4; 1.0

45±5; 24

4

12±1

r(CCUG)12–MBNL1

75±6; 1.4

29±3; 15

  1. *Secondary structures of the RNAs are provided in Supplementary Fig. S1.
  2. †The RNA and small molecule were pre-incubated followed by addition of MBNL1.
  3. ‡For studies with compound 1, the ligand was pre-incubated with MBNL1 followed by addition of RNA.
  4. §For studies with compound 2, the ligand was pre-incubated with the RNA followed by addition of MBNL1.
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