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  • RESEARCH HIGHLIGHT

Prime editing treats childhood neurological disease

By
  1. Sarah Crunkhorn
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Alternating hemiplegia of childhood (AHC) is a rare neurodevelopment disorder typically caused by mutations in ATP1A3, which encodes a subunit of the neuronal Na+/K+ ATPase protein complex. Although efforts to develop gene therapies are ongoing, there are currently no available disease-modifying treatments.

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Nature Reviews Drug Discovery 24, 742 (2025)

doi: https://doi.org/10.1038/d41573-025-00144-5

References

  1. Sousa, A. A. et al. In vivo prime editing rescues alternating hemiplegia of childhood in mice. Cell https://doi.org/10.1016/j.cell.2025.06.038 (2025)

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