Figure 1.

Cisterna Magna Delivery of scAAV9_hSOD1si in SOD1^G93A Mice Efficiently Reduces hSOD1 mRNA and Protein in Brain, Spinal Cord, and CSF

(A–F) qRT-PCR analysis shows depletion of hSOD1 mRNA in brainstem (A), cerebellum (B), striatum (C), cortex (D), and lumbar spinal cord (E) 4 weeks post-cisterna magna delivery of scAAV9_hSOD1si in post-natal day 1 (P1) SOD1^G93A mice. ELISA analysis shows a significant decrease in hSOD1 protein in CSF from the same group of mice (F). Data are presented as box and whiskers plot showing median, 25^th and 75^th percentiles, and maximum and minimum values. Data were analyzed by Student’s one-tailed t test (unpaired) (A–E, n ≥ 6; F, n ≥ 4). *p < 0.05, **p < 0.01, and ***p < 0.001.