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- W2754625379 abstract "Duchenne muscular dystrophy (DMD) is an X-linked genetic disorder caused predominantly by out-of-frame deletions in the DMD gene, resulting in absent or defective dystrophin protein. Exon-skipping technology has the potential to induce cellular machinery to ‘skip over’ a targeted exon and restore the reading frame, resulting in the production of internally truncated, but functional, dystrophin protein. WVE-210201 is an investigational stereopure antisense oligonucleotide (ASO) in development as a potential disease-modifying therapy targeting DMD exon 51 for the treatment of patients with DMD. WVE-210201 was developed using proprietary technologies that enable the production of nucleic acid therapeutics in which stereochemistry at each phosphorothioate position is precisely controlled. In vitro experiments were conducted to determine the stability, potency, and immune stimulatory effects of WVE-210201 and comparator ASOs. Gymnotic delivery of 10 µM of WVE-210201 to DMD Δ48–50 patient cells induced dystrophin protein restoration to a greater extent than comparator ASOs. There was a dose-dependent increase in skipped mRNA transcript that translated to truncated dystrophin protein restoration. These results were further confirmed in a second DMD patient-derived cell line (D52 cells). Furthermore, WVE-210201 demonstrated enhanced stability in mouse tissue homogenates relative to comparator ASOs, and did not activate toll-like receptor 9 (TLR9) in a human reporter cell line. These studies support further development of WVE-210201 as a potential disease-modifying therapy for the treatment of patients with DMD." @default.
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- W2754625379 date "2017-10-01" @default.
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- W2754625379 title "WVE-210201, an investigational stereopure oligonucleotide therapy for Duchenne muscular dystrophy, induces Exon 51 skipping and dystrophin protein restoration" @default.
- W2754625379 doi "https://doi.org/10.1016/j.nmd.2017.06.442" @default.
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