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PMO (phosphorodiamidate morpholino oligomer; splice-switching oligonucleotide)

Linked experiment: EXP001991

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 fields

RNA Type

PMO (phosphorodiamidate morpholino oligomer; splice-switching oligonucleotide)

RNA Payload Or Target

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

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).