Genome modification leads to phenotype reversal in human myotonic dystrophy type 1 induced pluripotent stem cell-derived neural stem cells

Guangbin Xia, Yuanzheng Gao, Shouguang Jin, S. H. Subramony, Naohiro Terada, Laura P W Ranum, Maurice S. Swanson, Tetsuo Ashizawa

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Medicine & Life Sciences