Fig. 5: Targeting new intronic donor site-activating ARFGEF1 mutants with YSC-004 using three different chemistries. | Experimental & Molecular Medicine

Fig. 5: Targeting new intronic donor site-activating ARFGEF1 mutants with YSC-004 using three different chemistries.

From: Tailored antisense oligonucleotides designed to correct aberrant splicing reveal actionable groups of mutations for rare genetic disorders

Fig. 5

a YSC-004 was designed to target the new donor site of AT1, a cryptic exon-activating mutant. The gel image shows the wild-type and aberrant transcripts (AT1) in the wild-type minigene, mutant minigene, mutant minigene following standard morpholino treatment (40 µM), and mutant minigene following control morpholino treatment (40 µM). b The gel image shows the effects of standard morpholino at different concentrations on the wild-type and aberrant transcripts (AT1) of the mutant minigene. The graph compares the wild-type and aberrant transcript expression levels (AT1) of the mutant minigene following ASO treatment to those of the wild-type minigene. c The gel image shows the effects of different concentrations of Vivo-morpholino on the wild-type and aberrant (AT1) transcript levels of the mutant minigene. The graph compares the wild-type and aberrant transcript (AT1) expression levels of the mutant minigene following ASO treatment to those of the wild-type minigene. d The gel image shows the effects of 2′MOE at different concentrations on the wild-type and aberrant transcripts (AT1) of the mutant minigene. The graph compares the wild-type and aberrant transcript (AT1) expression levels of the mutant minigene following ASO treatment to those of the wild-type minigene. e The summary table summarizes the outcomes of three different types of ASOs.

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