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SCO

Linked experiment: EXP002277

Linked peptide: DG9

Linked paper: DG9-conjugated morpholino rescues phenotype in SMA mice by reaching the CNS via a subcutaneous administration (2023)

RNA overview

RNA Type

SCO

Payload/target

SMN2 ISS-N1 (intron 7) splice-switching; promotes exon 7 inclusion

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

PMO; 18-mer morpholino (sequence identical to nusinersen target)