SCO

Linked experiment: EXP002442

Linked peptide: B-PMO 186

Linked paper: Cell-penetrating peptide-conjugated, splice-switching oligonucleotides mitigate the phenotype in Btk/Tec double deficient X-linked agammaglobulinemia model (2025)

RNA overview

RNA Type: SCO
Payload/target: SSO 186
Target gene/transcript: BTK pre-mRNA pseudoexon 4a / aberrant intron 4 pseudoexon inclusion

Sequence information

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

Chemical modifications

RNA modifications: phosphorodiamidate morpholino oligomer (PMO) SSO 186; peptide-conjugated