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PMO (splice-switching antisense morpholino)

Linked experiment: EXP002435

Linked peptide: DG9

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

RNA fields

RNA Type

PMO (splice-switching antisense morpholino)

RNA Payload Or Target

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

RNA Modifications

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