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Bi-specific splice-switching PMO oligonucleotides conjugated via a single peptide active in a mouse model of Duchenne muscular dystrophy

Paper ID

PAPER000330

Year

2015

DOI

10.1093/nar/gku1256

PMID

25468897

Journal

Nucleic Acids Research

Source URL

Experiments

• EXP001983 with Pip6a-PMO (Dmd exon23) single conjugate
• EXP001984 with Pip6a-PMO (Acvr2b exon5) single conjugate
• EXP001985 with Bi-specific D1 (one PMO on each Pip6a terminus)
• EXP001986 with Bi-specific D2 (both PMOs attached at Pip6a C-terminus; Acvr2b PMO click)
• EXP001987 with Bi-specific D3 (both PMOs attached at Pip6a C-terminus; Dmd PMO click)
• EXP001988 with Bi-specific D4 (C-terminal disulfide linkage for second PMO)
• EXP001989 with Bi-specific D2 (in vivo) (PMO (phosphorodiamidate morpholino oligomer; splice-switching oligonucleotide))
• EXP001990 with Bi-specific D3 (in vivo) (PMO (phosphorodiamidate morpholino oligomer; splice-switching oligonucleotide))
• EXP001991 with 1:1 cocktail of single Pip6a-PMOs (Dmd + Acvr2b) (in vivo) (PMO (phosphorodiamidate morpholino oligomer; splice-switching oligonucleotide))