Fig. 2: Efficacy, distribution, and persistence by Chol-HDO[PMO]. | Nature Communications

Fig. 2: Efficacy, distribution, and persistence by Chol-HDO[PMO].

From: Morpholino–RNA duplex exhibits robust, sustained, and safe steric-block antisense activity by intracerebroventricular and intrathecal injection

Fig. 2

a Dose–response curve of exon 23 skipping in the CNS following a single ICV injection of ssPMO (n = 3), 20-mer ssMOE (n = 3), or Chol-HDO[PMO] (n = 3 for the 5 nmol group, otherwise n = 4) (upper). Skipping potency was calculated from the fitted curves, and the fold change in potency is shown (lower). b Skipping activity of ssPMO (n = 3), 20-mer ssMOE (n = 4 for the 5-week group, otherwise n = 3), or Chol-HDO[PMO] (n = 4 for 2-week group, otherwise n = 3) at 20 nmol in six CNS regions at each time point. Data were collected 2, 5, and 9 weeks after a single ICV injection. c Exon 23 skipping rate in the CNS, liver, kidney, and quadriceps of rats 2 weeks after a single intrathecal injection of PBS (n = 3), 60 nmol ssPMO (n = 4), or Chol-HDO[PMO] (n = 4). All graphs display mean ± s.e.m.

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