Two studies highlight the evolution of antisense oligonucleotide therapy for amyotrophic lateral sclerosis, offering hope for an effective treatment.
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Acknowledgements
This work was supported in part by the Intramural Research Programs of the National Center for Advancing Translational Sciences and the National Institute on Aging, National Institutes of Health (Z01-AG000949-02).
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B.J.T. holds the US, Canadian and European patents on the clinical testing and therapeutic intervention for the hexanucleotide repeat expansion in C9orf72.
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Lopez, E.R., Borschel, W.F. & Traynor, B.J. New antisense oligonucleotide therapies reach first base in ALS. Nat Med 28, 25–27 (2022). https://doi.org/10.1038/s41591-021-01629-7
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DOI: https://doi.org/10.1038/s41591-021-01629-7
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