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Studies have shown that Pip6a-PMO effectively rescues the disease phenotype and improves the survival rate of severe SMA mice, showing strong efficacy in the central nervous system and peripheral tissues

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Ollivier, M

10.1016/S0076-6879(02)48630-2 Methods Enzymol

Then, to elucidate the potential mechanisms within the host, we performed transcriptome analysis on ear skin tissues from the model and LP-EVs intervention groups, which identified nearly 30,000 differentially expressed genes (Figure S6)