Development of single-stranded RNAsfor therapeutic gene knock-down

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This project will study key features of the triplet repeat RNA sequence associated with myotonic dystrophy type 1 and type 2. Specifically we will:1. Perform Super Resolution microscopy to confirm or refute the presence of CUG repeat fragments in the nuclear and cytoplasmic compartments of DM1 cell lines.2. Examine DM2 cell lines for similar fragments of CCUG repeats.3. Perform oligo-based capture experiments with subsequent analysis (TLC + Mass Spec) to determine whether fragments, if present, are subjected to base modification. 4. Use CRISPR Cas 9 to generate DM cell lines with inducible CUG and CCUG repeats.