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PMO (splice-switching antisense oligonucleotide)

Linked experiment: EXP002295

Linked peptide: Pip6a

Linked paper: Systemic peptide-mediated oligonucleotide therapy improves long-term survival in spinal muscular atrophy (2016)

RNA fields

RNA Type

PMO (splice-switching antisense oligonucleotide)

RNA Payload Or Target

SMN2 exon 7 (ISS-N1)

RNA Modifications

morpholino backbone (uncharged)