...successfully treated with a CRISPR gene editing therapy personalized to his genetic mutation. “Baby KJ” was diagnosed...Ahrens-Nicklas administered the first dose of his personalized genetic medicine (Musunuru et al., 2025).
Much... C., Rees, H. A., Packer, M. S., Badran, A. H., Bryson, D. I., & Liu, D. R. (2017). Programmable base ....2015.12.041
Musunuru, K., Grandinette, S. A., Wang, X., Hudson, T. R., Briseno, K., Berry, A. M., Hacker, J. L...Silverstein, R. A., Hille, L. T., Ogul, A. N., Robinson-Garvin, N. A., Small, J. C., McCague, S., Burke...https://doi.org/10.1056/NEJMoa2504747
Rees, H. A., Wilson, C., Doman, J. L., & Liu, D. R. (2019). Analysis..., Lapinaite, A., Newby, G. A., Thuronyi, B. W., Wilson, C., Koblan, L. W., Zeng, J., Bauer, D. E., Doudna...