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PUBLICATION

  • Jiang Y, Chen S, Hsiao S, Zhang H, Xie D, Wang ZJ, Ren W, Liu M, Liao J, Wu Y. Efficient and Safe In Vivo Treatment of Primary Hyperoxaluria Type 1 via LNP-CRISPR/Cas9-mediated glycolate oxidase disruption. Mol Ther. 2024 Oct 8:S1525-0016(24)00663-4. doi: 10.1016/j.ymthe.2024.10.003. Epub ahead of print. PMID: 39385468.

  • Fu, B., Liao, J., Chen, S., Li, W., Wang, Q., Hu, J., Yang, F., Hsiao, S., Jiang, Y., Wang, L., Chen, F., Zhang, Y., Wang, X., Li, D., Liu, M., &Wu, Y.2022. CRISPR-Cas9-mediated gene editing of the BCL11A enhancer for pediatric beta(0)/beta(0) transfusion-dependent beta-thalassemia.Nat Med, 28: 1573–1580.

  • Demirci, S., Zeng, J.,Wu, Y., Uchida, N., Shen, A. H., Pellin, D., Gamer, J., Yapundich, M., Drysdale, C., Bonanno, J., Bonifacino, A. C., Krouse, A. E., Linde, N. S., Engels, T., Donahue, R. E., Haro-Mora, J. J., Leonard, A., Nassehi, T., Luk, K., Porter, S. N., Lazzarotto, C. R., Tsai, S. Q., Weiss, M. J., Pruett-Miller, S. M., Wolfe, S. A., Bauer, D. E., & Tisdale, J. F. 2020. BCL11A enhancer-edited hematopoietic stem cells persist in rhesus monkeys without toxicity.J Clin Invest, 130(12): 6677-6687.

  • Zeng, J.,Wu, Y., Ren, C., Bonanno, J., Shen, A. H., Shea, D., Gehrke, J. M., Clement, K., Luk, K., Yao, Q., Kim, R., Wolfe, S. A., Manis, J. P., Pinello, L., Joung, J. K., & Bauer, D. E. 2020. Therapeutic base editing of human hematopoietic stem cells.Nat Med, 26(4): 535-541.

  • Zhang, X., Chen, L., Zhu, B., Wang, L., Chen, C., Hong, M., Huang, Y., Li, H., Han, H., Cai, B., Yu, W., Yin, S., Yang, L., Yang, Z., Liu, M., Zhang, Y., Mao, Z.,Wu, Y., Liu, M., & Li, D. 2020a. Increasing the efficiency and targeting range of cytidine base editors through fusion of a single-stranded DNA-binding protein domain.Nat Cell Biol, 22(6): 740-750.

  • Zhang, X., Zhu, B., Chen, L., Xie, L., Yu, W., Wang, Y., Li, L., Yin, S., Yang, L., Hu, H., Han, H., Li, Y., Wang, L., Chen, G., Ma, X., Geng, H., Huang, W., Pang, X., Yang, Z.,Wu, Y., Siwko, S., Kurita, R., Nakamura, Y., Yang, L., Liu, M., & Li, D. 2020b. Dual base editor catalyzes both cytosine and adenine base conversions in human cells.Nat Biotechnol, 38(7): 856-860.