Extended Data Fig. 8. The effect of ION363 on FUS expression and pathology in human lumbar spinal cord.

(a) Low-power immunohistochemical images of FFPE sections of lumbar spinal cord from a non-ALS control (left), ALS-FUS^P525L control patient (middle), and ION363-treated ALS-FUS^P525L patient (right) stained with an antibody against total FUS (FUS-Bethyl[400–450], top row), P525L-specific mouse monoclonal antibody reactive to FUS aggregates (middle row), and P525L-specific guinea pig antiserum (bottom row). Scale bar=100 µm. (b) Immunohistochemical staining of FFPE sections from lumbar spinal cord of a non-ALS control, ALS-FUS^P525L control patient, and ION363-treated ALS-FUS^P525L patient with P525L-specific guinea pig antiserum. Scale bar=20 µm.