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Third, there is evidence of successful restoration of aberrant mRNA splicing in non-NDD muscular conditions such as Spinal Muscular Atrophy (SMA) [89] and Duchenne Muscular Dystrophy (DMD) [90] through targeting splice junctions and cis-regulatory elements with ASOs [91, 92] (Fig

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The capsule formulation is designed for research examining the peptides activity when administered through the gastrointestinal route