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SCO

Linked experiment: EXP000741

Linked peptide: Myr-ApoE (peptide amphiphile)

Linked paper: Amphiphilic lipopeptide significantly enhances uptake of charge-neutral splice switching morpholino oligonucleotide in spinal muscular atrophy patient-derived fibroblasts (2017)

RNA overview

RNA Type

SCO

Payload/target

SMN2 exon 7 inclusion (splice correction)

Target gene/transcript

not reported

Sequence information

Sequence reported in source article

no

RNA sequence

not reported

Sense strand

Antisense strand

Chemical modifications

RNA modifications

phosphorodiamidate morpholino; 3'-maleimide functionalized for conjugation