Skip to main content

Thank you for visiting nature.com. You are using a browser version with limited support for CSS. To obtain the best experience, we recommend you use a more up to date browser (or turn off compatibility mode in Internet Explorer). In the meantime, to ensure continued support, we are displaying the site without styles and JavaScript.

NEUROGENOMICS

Translating amyotrophic lateral sclerosis genes into drug development leads

Although clear genetic connections to ALS were first established three decades ago, there has been negligible progress in the development of disease-modifying treatments for this disease. Despite tremendous unmet need, industry has often been largely baffled by a disease seemingly designed to thwart current effective drug development approaches. In the largest genetic study of ALS to date, van Rheenen and colleagues use sophisticated analyses to gain novel insights into its pathogenesis.

This is a preview of subscription content, access via your institution

Access options

Buy article

Get time limited or full article access on ReadCube.

$32.00

All prices are NET prices.

Fig. 1: ALS-associated genes and approved drugs.

References

  1. van Rheenen, W. et al. Nat. Genet. https://doi.org/10.1038/s41588-021-00973-1 (2021).

  2. Oakes, J. A. et al. Mol. Brain 10, 5 (2017).

    Article  Google Scholar 

  3. Kiernan, M. C. et al. Nat. Rev. Neurol. 17, 104–118 (2021).

    Article  Google Scholar 

  4. Phillips, T. et al. Curr. Protoc. Pharmacol. 69, 5.67.1–5.67.21 (2015).

    Article  Google Scholar 

  5. Richard, J. et al. Brain Res. 1607, 15–25 (2015).

    Article  CAS  Google Scholar 

  6. Lebedeva, I. V. et al. Cell Death Dis. 12, 770 (2021).

    Article  CAS  Google Scholar 

  7. Segeritz, C. J. et al. Hepatol. 69, 49–64 (2018).

    Article  Google Scholar 

  8. Prilutsky, D. et al. Trends Mol. Med. 20, 91–104 (2014).

    Article  CAS  Google Scholar 

Download references

Author information

Authors and Affiliations

Authors

Corresponding authors

Correspondence to Sunil Sahdeo or David B. Goldstein.

Ethics declarations

Competing interests

S.S. and D.B.G. are both employees of Actio Biosciences, a precision medicine biotechnology company. D.B.G. is a co-founder of Praxis Precision Medicines.

Rights and permissions

Reprints and Permissions

About this article

Verify currency and authenticity via CrossMark

Cite this article

Sahdeo, S., Goldstein, D.B. Translating amyotrophic lateral sclerosis genes into drug development leads. Nat Genet 53, 1624–1626 (2021). https://doi.org/10.1038/s41588-021-00981-1

Download citation

  • Published:

  • Issue Date:

  • DOI: https://doi.org/10.1038/s41588-021-00981-1

Search

Quick links

Nature Briefing

Sign up for the Nature Briefing newsletter — what matters in science, free to your inbox daily.

Get the most important science stories of the day, free in your inbox. Sign up for Nature Briefing