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Time: 4pm CET
Date: March 16, 2023
We use chemistry to improve the properties of nucleic acid therapeutics including antisense oligonucleotides, siRNAs, and genome editing approaches.
I will walk through a recent study we carried out trying to activate frataxin expression to treat Friedreich’s Ataxia. We did a lot of the traditional “right” controls but still found ourselves vulnerable to off-target effects. Finally, a combination of next-gen sequencing and gene editing proved that our effect was indirect.