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ASO

Linked experiment: EXP000916

Linked peptide: ApoE(140-150) CPP – thiol–CBT linkage (C4)

Linked paper: Thiol-Cyanobenzothiazole Ligation for the Efficient Preparation of Peptide-PNA Conjugates (2019)

RNA overview

RNA Type

ASO

Payload/target

SMN2 pre-mRNA splice correction (promote exon 7 inclusion; full-length SMN2 transcript)

Target gene/transcript

pre-

Sequence information

Sequence reported in source article

yes

RNA sequence

sense:ATTCACTTTCATAATGCTGG

Sense strand

ATTCACTTTCATAATGCTGG

Antisense strand

Chemical modifications

RNA modifications

PNA sequence 5'-ATTCACTTTCATAATGCTGG-3' with N-terminal miniPEG spacer and functional group for ligation; 5'-cysteine (PNA precursor used for thiol–CBT ligation)