IJMMS_2024v14n3

International Journal of Molecular Medical Science, 2024, Vol.14, No.3, 193-202 http://medscipublisher.com/index.php/ijmms 202 Romero Z., Lomova A., Said S., Miggelbrink A., Kuo C., Campo-Fernandez B., Hoban M., Masiuk K., Clark D., Long J., Sanchez J., Vélez M., Miyahira E., Zhang R., Brown D., Wang X., Kurmangaliyev Y., Hollis R., and Kohn D., 2019, Editing the sickle cell disease mutation in human hematopoietic stem cells: comparison of endonucleases and homologous donor templates, Molecular Therapy: the Journal of the American Society of Gene Therapy, 27(8): 1389-1406. https://doi.org/10.1016/j.ymthe.2019.05.014 PMid:31178391 PMCid:PMC6697408 Rosanwo T., and Bauer D., 2021, Editing outside the body: ex vivo gene-modification for β-hemoglobinopathy cellular therapy, Molecular Therapy: the Journal of the American Society of Gene Therapy, 29(11): 3163-3178. https://doi.org/10.1016/j.ymthe.2021.10.002 PMid:34628053 PMCid:PMC8571174 Uchida N., Li L., Nassehi T., Drysdale C., Yapundich M., Gamer J., Haro-Mora J., Demirci S., Leonard A., Bonifacino A., Krouse A., Linde N., Allen C., Peshwa M., Ravin S., Donahue R., Malech H., and Tisdale J., 2021, Preclinical evaluation for engraftment of CD34+ cells gene-edited at the sickle cell disease locus in xenograft mouse and non-human primate models, Cell Reports Medicine, 2(4): 100247. https://doi.org/10.1016/j.xcrm.2021.100247 PMid:33948577 PMCid:PMC8080237 Urnov F., 2021, The Cas9 hammer-and sickle: a challenge for genome editors, The CRISPR Journal, 4(1): 6-13. https://doi.org/10.1089/crispr.2021.29120.fur PMid:33616446 Wilkinson A., Dever D., Baik R., Camarena J., Hsu I., Charlesworth C., Morita C., Nakauchi H., and Porteus M., 2020, Cas9-AAV6 gene correction of beta-globin in autologous HSCs improves sickle cell disease erythropoiesis in mice, Nature Communications, 12: 686. https://doi.org/10.1101/2020.10.13.338319 Zarghamian P., Klermund J., and Cathomen T., 2023, Clinical genome editing to treat sickle cell disease-a brief update, Frontiers in Medicine, 9: 1065377. https://doi.org/10.3389/fmed.2022.1065377 PMid:36698803 PMCid:PMC9868311 Zeng J., Wu Y., Wu Y., Ren C., Bonanno J., Shen A., Shea D., Gehrke J., Clement K., Luk K., Yao Q., Kim R., Kim R., Wolfe S., Manis J., Pinello L., Joung J., and Bauer D., 2020, Therapeutic base editing of human hematopoietic stem cells, Nature Medicine, 26: 535-541. https://doi.org/10.1038/s41591-020-0790-y PMid:32284612 PMCid:PMC7869435

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