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Thomsen, G., Burghes, A.H.M., Hsieh, C., Do, J., Chu, B.T.T., Perry, S., et al. (2021) Biodistribution of Onasemnogene Abeparvovec DNA, mRNA and SMN Protein in Human Tissue. Nature Medicine, 27, 1701-1711.
https://doi.org/10.1038/s41591-021-01483-7

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