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ASO

Linked experiment: EXP000272

Linked peptide: HA2-ApoE(133–150)

Linked paper: Endosomal escape cell-penetrating peptides significantly enhance pharmacological effectiveness and CNS activity of systemically administered antisense oligonucleotides (2021)

RNA overview

RNA Type

ASO

Payload/target

SMN2 exon-7 splice correction

Target gene/transcript

not reported

Sequence information

Sequence reported in source article

yes

RNA sequence

antisense:ATTCACTTTCATAATGCTGG

Sense strand

Antisense strand

ATTCACTTTCATAATGCTGG

Chemical modifications

RNA modifications

Phosphorodiamidate morpholino