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cDNA was synthesized and RNA-seq libraries were constructed using the TruSeq RNA Sample Prep kit from Illumina

For the exon skipping efficacy test, BC-derived primary cells (3 10 cells per well) were seeded in a 6-well plate, and the most promising AON was selected by transfecting 300 nM AON using Lipofectamine RNAiMAX reagent (Invitrogen, Thermo Fisher Scientific, #13778075) according to the manufacturers instructions

It can protect against further damage

Cells that overexpressed HGT1 were unable to grow on minimal medium plates that contained GSH at concentrations as low as 20 M (Figure 1A)

Patients typically get relief the day of the injection