Madaiah Puttaraju

Author Correction: Systematic screening identifies therapeutic antisense oligonucleotides for Hutchinson–Gilford progeria syndrome Nature Medicine (2021)Cite this article Subjects The Original Article was published on 11 March 2021 Correction to: Nature Medicine https://doi.org/10.1038/s41591-021-01262-4, published online 11 March 2021. In the version of this article initially published, the source of lonafarnib was not provided.

Abstract Hutchinson–Gilford progeria syndrome (HGPS) is a rare, invariably fatal childhood premature aging disorder caused by a pre-messenger RNA (mRNA) splicing defect in the LMNA gene. We used combined in vitro screening and in vivo validation to systematically explore the effects of target sequence, backbone chemistry and mechanism of action to identify optimized antisense oligonucleotides (ASOs) for therapeutic use in HGPS.