Base editing-mediated splicing correction therapy for spinal muscular atrophy

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Fig. 1: Base editing-mediated conversion of exonic splicing silencers rescues SMA.


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This work was supported by grants from the National Natural Science Foundation of China (81771230, 31522037, U1905210, and 31922048), the Joint Funds for the Innovation of Science and Technology of Fujian Province (2017Y9094 and 2018Y9082), the National Key Clinical Specialty Discipline Construction Program, the Key Clinical Specialty Discipline Construction Program of Fujian, the National Science and Technology major project (2017YFC1001302), the Shanghai City Committee of Science and Technology project (16JC1420202), and the Agricultural Science and Technology Innovation Program.

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W.-J.C., H.Y. and E.Z. designed this study. X.L., H.C., Y.-Q.L., S.H. and X.H. wrote the initial manuscript and constructed the figures. W.-J.C., H.Y., E.Z., N.W. and L.M. contributed to the editing of the manuscript, figures, and tables. X.L., H.C., Y.-Q.L., S.H. and J.-J.L. performed genome editing in HEK293T cells, SMA mESCs, and SMA iPSCs. X.L. and L.-L.L. performed experiments on motor neurons. X.H., S.H. and Y.G. performed experiments on animals. W.Y. and E.Z. transferred embryos. Z.W. performed the data analysis regarding high-throughput sequencing. H.C. and S.H. performed the statistical analysis. All authors have read and approved the final manuscript.

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Correspondence to Erwei Zuo or Hui Yang or Wan-Jin Chen.

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The authors declare no competing interests.

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Lin, X., Chen, H., Lu, Y. et al. Base editing-mediated splicing correction therapy for spinal muscular atrophy. Cell Res 30, 548–550 (2020).

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