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The designed ASOs mask the dysfunctional exon in the pre-mRNA, resulting in the exon being spliced out of the final mRNA transcriptthus restoring the reading frame and producing a partially functioning copy of the dystrophin protein [92, 97] (Fig

The cream format helps these molecules penetrate deeper into the skin layers, where they can work their magic more effectively than lighter formulations

While oral bioavailability may be lower compared to injections, it offers systemic benefits and convenience

Neuvonen, Clinical trial with an experimental taurine derivative, taltrimide, in epileptic patients, Epilepsia 27(1) (1986) 87-90

Simply combining the presence of E

Injection site rotation is important both for comfort and for maximizing local benefits