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

Peptide ID

PEPTIDE001528

Raw sequence

RXRRBRRXRYQFLIRXRBRXRB

Clean backbone

RAHXRRBLARRAHXRYQFLIRAHXRBLARAHXRBLA

Tokenized backbone

R-AHX-R-R-BLA-R-R-AHX-R-Y-Q-F-L-I-R-AHX-R-BLA-R-AHX-R-BLA

Sequence engineering

Stereochemistry

Modifications

Pip6a is an arginine-rich CPP (Pip family). In the paper, X = aminohexanoic acid spacer; B = β-alanine; Bpg = bis-homopropargylglycine (alkyne).

Sequence length

36

Noncanonical residues

Experiments

• EXP001991 from Bi-specific splice-switching PMO oligonucleotides conjugated via a single pepti…