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.

  • News & Views
  • Published:

Epilepsy

‘On-demand’ gene therapy for epilepsy

Subjects

Gene therapies show promise for treating epilepsy, but most strategies target cells across an entire brain region rather than selecting pathologically hyperexcited neurons. Researchers have now developed a conditional gene therapy strategy that downregulates firing activity only in neurons that are pathologically overactive and switches off when brain circuit activity has returned to baseline.

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

Access options

Buy this article

Prices may be subject to local taxes which are calculated during checkout

References

  1. French, J. A. Refractory epilepsy: one size does not fit all. Epilepsy Curr. 6, 177–180 (2006).

    Article  PubMed  PubMed Central  Google Scholar 

  2. Striano, P. & Minassian, B. A. From genetic testing to precision medicine in epilepsy. Neurotherapeutics 17, 609–615 (2020).

    Article  PubMed  PubMed Central  Google Scholar 

  3. Zhang, L. & Wang, Y. Gene therapy in epilepsy. Biomed. Pharmacother. 143, 112075 (2021).

    Article  CAS  PubMed  Google Scholar 

  4. Qiu, Y. et al. On-demand cell-autonomous gene therapy for brain circuit disorders. Science 378, 523–532 (2022).

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  5. Liu, X. et al. Optogenetic stimulation of a hippocampal engram activates fear memory recall. Nature 484, 381–385 (2012).

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  6. Tønnesen, J., Sørensen, A. T., Deisseroth, K., Lundberg, C. & Kokaia, M. Optogenetic control of epileptiform activity. Proc. Natl Acad. Sci. USA 106, 12162–12167 (2009).

    Article  PubMed  PubMed Central  Google Scholar 

  7. Krook-Magnuson, E., Armstrong, C., Oijala, M. & Soltesz, I. On-demand optogenetic control of spontaneous seizures in temporal lobe epilepsy. Nat. Commun. 4, 1376 (2013).

    Article  PubMed  Google Scholar 

  8. Lieb, A. et al. Biochemical autoregulatory gene therapy for focal epilepsy. Nat. Med. 24, 1324–1329 (2018).

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  9. Walker, M. C. & Kullmann, D. M. Optogenetic and chemogenetic therapies for epilepsy. Neuropharmacology 168, 107751 (2020).

    Article  CAS  PubMed  Google Scholar 

Download references

Author information

Authors and Affiliations

Authors

Corresponding author

Correspondence to Pasquale Striano.

Ethics declarations

Competing interests

The authors declare no competing interests.

Rights and permissions

Reprints and permissions

About this article

Check for updates. Verify currency and authenticity via CrossMark

Cite this article

Striano, P., Benfenati, F. ‘On-demand’ gene therapy for epilepsy. Nat Rev Neurol 19, 130–131 (2023). https://doi.org/10.1038/s41582-022-00761-3

Download citation

  • Published:

  • Issue Date:

  • DOI: https://doi.org/10.1038/s41582-022-00761-3

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