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. 2015 Oct 12;112(43):E5863–E5872. doi: 10.1073/pnas.1509758112

Fig. 6.

Fig. 6.

Systemic ASO restores SMN RNA (A–D) and protein (E–H) levels in tissues including the spinal cord. Late administration increases SMN in muscles but not spinal cord. (A and B) SMN2 mRNA quantification in the diaphragm (A) and spinal cord (B) of PND 60 mutants treated with vehicle or ASO at PND 10. The ratio of full-length (including exon 7) SMN2 transcripts to SMN2 transcripts lacking exon 7 (δ7) is shown. ASO-treated mutants had higher full-length SMN2 expression in both tissues (Student t test, n = 6 per treatment group). (C and D) SMN2 mRNA quantification in the diaphragm (C) and spinal cord (D) of PND 60 mutants. ASO treatment specifically increased full-length SMN mRNA levels in the diaphragm but not in the spinal cord (Student t test, n = 6 per treatment group). (E–H) SMN protein quantification by ELISA at PND 25 in untreated mutants (n = 10) or mutants treated at PND 10 with vehicle (n = 6) or ASO (n = 5). ASO treatment increased SMN protein in the heart only (Student t test between vehicle- and ASO-treated mutants). (H) SMN protein quantification in peripheral blood mononuclear cells (PBMCs) did not detect any increase following PND 10 administration of vehicle (n = 5) or ASO (n = 5). Measurements were performed at PND 25, 30, and 35.