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Correction of theHEXA1278insTATC mutation that causes Tay-Sachs disease in a HEK293T cell line model previously engineered to harbor the mutation (left) and in patient-derived fibroblasts (right). n = 3 independent replicates were used for the HEK293T cell line model. n = 2 independent replicates were used for the patient-derived fibroblasts. For B-D, the DNA substrate is green, RNA substrate is yellow, residues mutated following PANCE in the v1 circuit are blue, residues mutated following PANCE in the v2 circuit are red, and residue mutated following PANCE in the v3 circuit is orange. For (E–H), bars show the mean value for the specified number of replicates, and dots show individual replicate values. See alsoFigure S3.
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