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PMO (splice-switching morpholino)

Linked experiment: EXP000800

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 fields

RNA Type

PMO (splice-switching morpholino)

RNA Payload Or Target

SMN2 exon 7 inclusion (splice correction)

RNA Modifications

phosphorodiamidate morpholino; 3'-maleimide functionalized for conjugation