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SCO

Linked experiment: EXP001839

Linked peptide: 1:1 cocktail of single Pip6a-PMOs (Dmd + Acvr2b) (in vivo)

Linked paper: Bi-specific splice-switching PMO oligonucleotides conjugated via a single peptide active in a mouse model of Duchenne muscular dystrophy (2015)

RNA overview

RNA Type

SCO

Payload/target

Dmd exon23 + Acvr2b exon5 splice switching in TA muscle (co-administered as separate conjugates)

Target gene/transcript

not reported

Sequence information

Sequence reported in source article

yes

RNA sequence

sense:GGCCAAACCTCGGCTTACCTGAAATa

Sense strand

GGCCAAACCTCGGCTTACCTGAAATa

Antisense strand

Chemical modifications

RNA modifications

Dmd exon23 PMO: 5′-GGCCAAACCTCGGCTTACCTGAAAT-3′; Acvr2b exon5 PMO: 5′-GCCTCGTTTCTCGGCAGCAATGAAC-3′ (Gene Tools; standard PMO with 3′ secondary amine; some functionalized with 3′ azido or 3′ NPys for conjugation).