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splice-switching PMO (morpholino)

Linked experiment: EXP001491

Linked peptide: ApoE (co-treated with VPA)

Linked paper: Combination of valproic acid and morpholino splice-switching oligonucleotide produces improved outcomes in spinal muscular atrophy patient-derived fibroblasts (2017)

RNA fields

RNA Type

splice-switching PMO (morpholino)

RNA Payload Or Target

SMN2 intron 7 ISS-N1 (splice-switching to promote exon 7 inclusion); PMO sequence: ATTCACTTTCATAATGCTGG (ISS-N1 targeting 20-mer PMO; 5′-ATTCACTTTCA-TAATGCTGG-3′)

RNA Modifications

PMO chemistry; 3′-maleimide functionalization for peptide conjugation