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Comment
. 2024 Oct 4;386(6717):24-25.
doi: 10.1126/science.ads5951. Epub 2024 Oct 3.

Regulators, mount up

Yi Zeng  1 , Aaron D Gitler  1
Affiliations
Comment

Regulators, mount up

Yi Zeng et al. Science. .

Abstract

Cryptic exons enable delivery of therapies only to sick neurons in a motor neuron disease.

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  • Creation of de novo cryptic splicing for ALS and FTD precision medicine.
    Wilkins OG, Chien MZYJ, Wlaschin JJ, Barattucci S, Harley P, Mattedi F, Mehta PR, Pisliakova M, Ryadnov E, Keuss MJ, Thompson D, Digby H, Knez L, Simkin RL, Diaz JA, Zanovello M, Brown AL, Darbey A, Karda R, Fisher EMC, Cunningham TJ, Le Pichon CE, Ule J, Fratta P. Wilkins OG, et al. Science. 2024 Oct 4;386(6717):61-69. doi: 10.1126/science.adk2539. Epub 2024 Oct 3. Science. 2024. PMID: 39361759 Free PMC article.

References

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