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ASO

Linked experiment: EXP000510

Linked peptide: BPP9

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

RNA overview

RNA Type

ASO

Payload/target

SMN2 exon-7 inclusion (ISS-N1)

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 backbone; Cy7 label in imaging studies (subset)