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Antisense approach slows progeria

  1. M. Teresa Villanueva
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Hutchinson–Gilford progeria syndrome (HGPS) is a disorder caused by single-nucleotide mutations in the lamin A gene (LMNA) and characterized by accelerated aging, cardiovascular defects and premature death. Currently the only treatment option is the very recently approved farnesyltransferase inhibitor, lonafarnib, but is has important side effects that can worsen the condition. Now, two articles in Nature Medicine describe the development of antisense oligonucleotides (ASOs) that target two different exons of LMNA, can successfully decrease both progerin transcripts and protein levels and can extend the lifespan of a mouse model of HGPS.

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Nature Reviews Drug Discovery 20, 343 (2021)

doi: https://doi.org/10.1038/d41573-021-00056-0

References

  1. Erdos, M. R. et al. A targeted antisense therapeutic approach for Hutchinson–Gilford progeria syndrome. Nat. Med. https://doi.org/10.1038/s41591-021-01274-0 (2021).

  2. Puttaraju, M. et al. Systematic screening identifies therapeutic antisense oligonucleotides for Hutchinson–Gilford progeria syndrome. Nat. Med. https://doi.org/10.1038/s41591-021-01262-4 (2021).

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