WVE-210201, an investigational stereopure oligonucleotide therapy for Duchenne muscular dystrophy, induces Exon 51 skipping and dystrophin protein restoration


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.


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