Extended Data Fig. 7: Cortical distribution of ION363 and its effect on total protein levels of RBPs.
(a) Anti-ASO immunohistochemical staining of FFPE sections of motor cortex (BA4) from a non-ALS control (left) and ION363-treated ALS-FUSP525L patient (right). Scale bar=200 µm at 4x and 20 µm at 40x. (b) Immunoblot of sarkosyl solubility fractionation of brain stem samples from a non-ALS control, ALS-FUSP525L control patient, and ION363-treated ALS-FUSP525L patient. Sol = soluble (in hypotonic buffer), SS = sarkosyl soluble (in 1% sarkosyl and high salt), and SI = sarkosyl insoluble fractions. (c) Representative immunoblot of brainstem tissue from a non-ALS control, ALS-FUSP525L control patient, and ION363-treated ALS-FUSP525L patient probed with antibodies against RBPs.
