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new therapeutic small-molecules and antisense oligonucleotides targeting mutated RNA molecules containing expanded repeats. A brief project description can be found at: https://ibmib.web.amu.edu.pl/mab
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with—and drawing on the expertise of—Prof. Sobczak’s group, ASOs will be designed to target selected regulatory elements in order to reduce DMPK/CNBP expression or increase the expression of MBNL genes
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