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PMO (antisense oligonucleotide)

Linked experiment: EXP000529

Linked peptide: BPP4

Linked paper: Efficient systemic CNS delivery of a therapeutic antisense oligonucleotide with a blood-brain barrier-penetrating ApoE-derived peptide (2024)

RNA fields

RNA Type

PMO (antisense oligonucleotide)

RNA Payload Or Target

SMN2 exon-7 inclusion (ISS-N1)

RNA Modifications

PMO backbone; Cy7 label in imaging studies (subset)