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VPA Treatment Rescues the NMJ Pathology in SMA Patient-Derived Cell Cultures
(A) Representative images of NMJ-LSs formed with or without VPA. Yellow arrows indicate abnormal NF accumulation.
(B and C) Quantitative immunocytochemical analysis of the α-BTX area after VPA treatment (1 mM) (means ± SEM, n = 3, Student’s t test).
(D) The mRNA levels of SMN-FL and SMN-Δ7 in SMA-iPSC MNs (means ± SEM, n = 3, Student’s t test). See also Figure S3 and Tables S1 and S2.
