Genomic splice-donor disruption rescues systemic disease in progeria

Wei Jin Jing-Chun Ma Hao Yuan Yu Zou Zi-Xuan Zhang Jin-Hang Zuo Sheung-Kin-Ken Wong Tuo Li Xiao-Mei Lu Zhong-Jun Zhou

Sep 2026 · bioRxiv · 0 citations · 29 references

Biology

TL;DR

These findings establish pathogenic splice signals as genomic targets for sustained suppression of toxic isoforms while retaining canonical gene output and establish pathogenic splice signals as genomic targets for sustained suppression of toxic isoforms while retaining canonical gene output.

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