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MOTOR NEURON DISEASE

Moving towards genomic therapy for amyotrophic lateral sclerosis

Nature Reviews Neurology volume 16, page 460 (2020)Cite this article

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Superoxide dismutase 1 (SOD1) gene mutations account for up to 20% of cases of familial amyotrophic lateral sclerosis (ALS), and this gene could represent an important therapeutic target. Two new reports in The New England Journal of Medicine provide early indications that knockdown of SOD1 expression is feasible and warrants further exploration in people with SOD1 ALS.

The first study used the antisense oligonucleotide (ASO) tofersen to target SOD1. “ASOs bind to the target mRNA and activate RNAse H, which degrades the mRNA,” explains lead author Timothy Miller. “As mutations in SOD1 cause a toxic gain of function, lowering the levels of SOD1 protein is predicted to be therapeutic.”

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References

Original articles

  • Miller, T. et al. Phase 1–2 trial of antisense oligonucleotide tofersen for SOD1 ALS. N. Engl. J. Med. 383, 109–119 (2020)

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  • Mueller, C. et al. SOD1 suppression with adeno-associated virus and microRNA in familial ALS. N. Engl. J. Med. 383, 151–158 (2020)

    Article  CAS  Google Scholar 

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  1. Nature Reviews Neuroscience http://www.nature.com/nrn/

    Heather Wood

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  1. Heather Wood
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Correspondence to Heather Wood.

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Wood, H. Moving towards genomic therapy for amyotrophic lateral sclerosis. Nat Rev Neurol 16, 460 (2020). https://doi.org/10.1038/s41582-020-0393-8

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  • DOI: https://doi.org/10.1038/s41582-020-0393-8

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