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Base editing treats SMA in mice

  1. M. Teresa Villanueva
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Spinal muscular atrophy (SMA), a progressive disease that affects motor neurons and the leading genetic cause of infant mortality, is caused by the loss or mutation of the gene encoding survival motor neuron (SMN), SMN1. Three drugs have recently been approved for the treatment of SMA, but their effect is transient or may wane and can require difficult redosing. Writing in Science, David Liu and colleagues describe a novel approach to edit SMN2, a near copy of SMN1, and restore levels of SMN. A single injection rescued motor function and significantly enhanced lifespan in mouse models of SMA.

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Nature Reviews Drug Discovery 22, 353 (2023)

doi: https://doi.org/10.1038/d41573-023-00057-1

References

  1. Arbab, M. et al. Base editing rescue of spinal muscular atrophy in cells and in mice. Science https://doi.org/10.1126/science.adg6518 (2023)

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