mRNA Degradation as a Therapeutic Solution for Mucopolysaccharidosis Type IIIC: Use of Antisense Oligonucleotides to Promote Downregulation of Heparan Sulfate Synthesis.

Although not statistically significant, a decreasing trend is observed when comparing the HS levels of the samples treated with the scramble ASO with the cells transfected with the gapmer G3 ( Figure 4 ).

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