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ASO

Linked experiment: EXP001577

Linked peptide: Pip6a-PMO-CAG7

Linked paper: Peptide-conjugated oligonucleotides evoke long-lasting myotonic dystrophy correction in patient-derived cells and mice (2019)

RNA overview

RNA Type

ASO

Payload/target

CUGexp transcripts in HSA-LR mouse (CAG7 PMO antisense)

Target gene/transcript

sense

Sequence information

Sequence reported in source article

yes

RNA sequence

sense:CAGCAGCAGCAGCAGCAGCAG

Sense strand

CAGCAGCAGCAGCAGCAGCAG

Antisense strand

Chemical modifications

RNA modifications

RNase H–inactive phosphorodiamidate morpholino oligomer; CAG7 sequence (or reverse-sequence control for Pip6a-Ctrl).