ASO

Linked experiment: EXP000503

Linked peptide: BPP3

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)