Fig. 4.
Pip6a-PMO administration in unaffected adult mice harboring the human SMN2 allele (Smn1tm1Hung/WT;SMN2tg/tg). (A–C) Quantitative RT-PCR from tissues of mice administered 18 mg/kg, i.v. 2 d apart. Tissues from brain (A), spinal cord (B), and peripheral skeletal muscles and liver (C) were harvested 7 d postadministration. In all tissues, FLSMN2 expression was significantly increased over saline-treated mice. (D) Protein expression of SMN in tissues. Separate tissues of spinal cord and skeletal muscles were combined. SMN protein was analyzed with Western blots for human SMN and mouse β-tubulin. Data are represented as mean ± SEM (*P ≤ 0.005; **P ≤ 0.001; ***P ≤ 0.0001 by Student’s t test in comparison to saline-treated mice).