Dystrophia myotonica type 1 (DM1) results from
nuclear sequestration of splicing factors by a messenger RNA (mRNA)
harboring a large (CUG)n repeat array
transcribed from the causal (CTG)n DNA
amplification. Several compounds were previously shown to bind the
(CUG)n RNA and release the splicing factors.
We now investigated for the first time the interaction of an aliphatic
polycarbonate carrying guanidinium functions to DM1 DNA/RNA model
probes by affinity capillary electrophoresis. The apparent association
constants (Ka) were in the range described
for reference compounds such as pentamidine. Further macromolecular
engineering could improve association specificity. The polymer presented
no toxicity in cell culture at concentrations of 1.6–100.0
μg/mL as evaluated both by MTT and real-time monitoring xCELLigence
method. These promising results may lay the foundation for a new branch
of potential therapeutic agents for DM1.
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