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    Correction/mutation of acid α-D-glucosidase gene by modified single-stranded oligonucleotides: in vitro and in vivo studies

    Deficiency in acid α-D-glucosidase results in Pompe's disease. Modified single-stranded oligonucleotide (ODN) was designed to correct the acid α-D-glucosidase gene with a C1935 → A (Asp → Glu) point mutation whic...

    I-L Lu, C-Y Lin, S-B Lin, S-T Chen, L-Y Yeh, F-Y Yang, L-C Au in Gene Therapy (2003)