Back to browse

SCO

Linked experiment: EXP002444

Linked peptide: Pip6a-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 187

Target gene/transcript

BTK pre-mRNA pseudoexon 4a / aberrant intron 4 pseudoexon inclusion

Sequence information

Sequence reported in source article

yes

RNA sequence

g*a*g*a*c*u*u*a*c*c*a*c*u*u*c*c*u*u*

Sense strand

Antisense strand

G*A*G*A*C*U*U*A*C*C*A*C*U*U*C*C*U*U*

Chemical modifications

RNA modifications

phosphoryl guanidine oligo-2′-O-methylribonucleotide (PGO) SSO 187; peptide-conjugated