Base editors can restore the expression of survival motor neuron protein to therapeutically beneficial levels in animal and cell models of spinal muscular atrophy.
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P.M. is a scientific co-founder of Navega Therapeutics, Boundless Biosciences, Shape Therapeutics and Engine Biosciences. The terms of these arrangements have been reviewed and approved by the University of California, San Diego, in accordance with its conflict-of-interest policies.
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Portell, A., Mali, P. Mutation corrections in spinal muscular atrophy. Nat. Biomed. Eng 8, 111–113 (2024). https://doi.org/10.1038/s41551-023-01166-3
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DOI: https://doi.org/10.1038/s41551-023-01166-3