Figure 2.
2′-O-Methyl bifunctional RNAs increase survival motor neuron (SMN) protein and gem number in patient fibroblasts. (a) Spinal muscular atrophy (SMA) patient fibroblasts (3,813 cells) were transiently transfected with 100 ng of hTra2β1-ISS-N1 and SF2/ASF-ISS-N1 2′-O-methyl bifunctional RNAs for 48 hours. Representative cells after immunofluorescence (IMF) staining. 3,813 cells are shown in the top row as positive control staining (b) 500 3,813 cells were randomly counted (n = 3) and total gem number determined. ISS-N1, intronic splicing silencer N1.