SCO

Linked experiment: EXP001385

Linked peptide: (N)-VPA-ApoE

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

RNA overview

RNA Type: SCO
Payload/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′)
Target gene/transcript: ISS-N1

Sequence information

Sequence reported in source article: yes
RNA sequence: antisense:ATTCACTTTCATAATGCTGG
Sense strand
Antisense strand: ATTCACTTTCATAATGCTGG

Chemical modifications

RNA modifications: PMO chemistry; 3′-maleimide functionalization for peptide conjugation