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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...