Population
Co-transfection model and DM1 fibroblasts
Design
Preclinical
Authors
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Foci and splicing defects are separable in DM1 models; leaves open whether foci drive pathogenesis and warrants validation before targeting.
The study demonstrates that in myotonic dystrophy type I, the formation of RNA foci and the disruption of MBNL1-regulated splicing are distinct and separable events.
Ho et al. (2005) studied this question.
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