Efficient systemic cellular uptake of therapeutics is an important determinant for the success of future clinical trials in DM1, because all known molecular targets, e.g., expanded (CUG)n/(CAG)n transcripts or CELF1 and MBNL1 proteins that bind to these repeat-containing RNAs [44] are located intracellularly. The gene discussed is MBNL1; the disease is myotonic dystrophy type 1.