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OTS Rare Disease N-of-1+ Briefing Document, Jan 2020

This briefing document aims to outline the current state of the art of oligonucleotide therapies for those planning to develop individualized therapies for patients with very rare diseases or mutations. The focus is on approved modalities and tissues for which good delivery of oligonucleotides has been confirmed in humans. We believe development of individualized therapies should build on those approaches.



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