Repeat length as a key determinant for disease severity and antisense oligonucleotide activity in myotonic dystrophy type 1.

Mol Ther Methods Clin Dev · 2025 · PMC12221585 · PMID 40606545

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a clear trendno p-value reported

More importantly, free nuclear MBNL1 abundance showed a clear trend in relation to repeat length, with longer repeat lengths corresponding to lower levels of available nuclear MBNL1 protein in the cell ( Figure 6 C).

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a marked trendno p-value reported

NFIX ex 7 missplicing was less distinct, but also here, a marked trend was observed between repeat length and exon inclusion levels.

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