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SCO

Linked experiment: EXP002443

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 186

Target gene/transcript

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

Sequence information

Sequence reported in source article

yes

RNA sequence

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

Sense strand

Antisense strand

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

Chemical modifications

RNA modifications

phosphoryl guanidine oligo-2′-O-methylribonucleotide (PGO) SSO 186; peptide-conjugated