Splice modulating antisense oligonucleotides restore some acid-alpha-glucosidase activity in cells derived from patients with late-onset Pompe disease

May Thandar Aung-Htut, Kristin A. Ham, Michel Tchan, Russell Johnsen, Frederick J. Schnell, Sue Fletcher, Steve D. Wilton

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8 Citations (Scopus)

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Biochemistry, Genetics and Molecular Biology

Immunology and Microbiology