Thomsen, G., Burghes, A. H. M., Hsieh, C., Do, J., Chu, B. T. T., Perry, S., Barkho, B., Kaufmann, P., Sproule, D. M., Feltner, D. E., Chung, W. K., McGovern, V. L., Hevner, R. F., Conces, M., Pierson, C. R., Scoto, M., Muntoni, F., Mendell, J. R., & Foust, K. D. (2021). Biodistribution of onasemnogene abeparvovec DNA, mRNA and SMN protein in human tissue. Nature Medicine, 27(10), 1701–1711. https://doi.org/10.1038/s41591-021-01483-7