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Article

Control of backbone chemistry and chirality boost oligonucleotide splice switching activity

7 Jan 2022

Abstract excerpt

Although recent regulatory approval of splice-switching oligonucleotides (SSOs) for the treatment of neuromuscular disease such as Duchenne muscular dystrophy has been an advance for the splice-switching field, current SSO chemistries have shown limited clinical benefit due to poor pharmacology. To overcome limitations of existing technologies, we engineered chimeric stereopure oligonucleotides with...

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PMID: 35061895
DOI: 10.1093/nar/gkac018

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