Antisense oligonucleotides that target lamin A/C pre-mRNA decrease both progerin transcript levels and progerin protein levels and extend lifespan in an animal model of Hutchinson–Gilford progeria syndrome.
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Revêchon, G., Whisenant, D. & Eriksson, M. Splice-inhibition therapy targets progeria. Nat Med 27, 377–379 (2021). https://doi.org/10.1038/s41591-021-01267-z
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DOI: https://doi.org/10.1038/s41591-021-01267-z