ASO

Linked experiment: EXP002142

Linked peptide: Pip6a

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

RNA overview

RNA Type: ASO
Payload/target: SMN2 exon 7 (ISS-N1)
Target gene/transcript: not reported

Sequence information

Sequence reported in source article: yes
RNA sequence: sense:ATTCACTTTCATAATGCTGG
Sense strand: ATTCACTTTCATAATGCTGG
Antisense strand

Chemical modifications

RNA modifications: morpholino backbone