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Unveiling sequence-agnostic mixed-chemical modification patterns for splice-switching oligonucleotides using the NATURA platform.

Tabaglio T, Agarwal T, Cher WY, Ow JR, Chew AK, Sun PYQ, Reddy Gurrampati RS, Lu H, Naidu P, Ng HK, Le Guezennec X, Ng SY, Lakshmanan M, Guccione E, Wee KB
Mol Ther Nucleic Acids. 2025 Jan 7;36(1):102422. doi: 10.1016/j.omtn.2024.102422. eCollection 2025 Mar 11. (Link opens in a new window) PubMed (Link opens in a new window) Article

Plasmids from Article

ID Plasmid Purpose
226553pRP.ABCA4 c.5461-10TMinigene encoding the ABCA4 c.5461-10T mutation
226554pRP.ATM c.7865C>TMinigene encoding the ATM c.7865C>T mutation
226555pRP.USH2AMinigene encoding USH2A exon 13 and surrounding introns/exons
226556pRP.CEP290 c.2991+1655A>GMinigene encoding the CEP290 c.2991+1655A>G mutation
226557pC2.4NATURA gene encoding plasmid

Antibodies from Article