ASO (PMO)

Linked experiment: EXP000251

Linked peptide: HA2

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

RNA fields

RNA Type: ASO (PMO)
RNA Payload Or Target: SMN2 exon-7 splice correction
RNA Modifications: Phosphorodiamidate morpholino