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Site-Specific Gene Editing of Human Hematopoietic Stem Cells for X-Linked Hyper-IgM Syndrome
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“More precise methods of gene editing, such as base editing and prime editing, are being developed to correct individual mutations causing IEIs”
From Ex vivo modification of hematopoietic stem and progenitor cells for gene therapy (2025) · cites this paper as [52]
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Cited by
Ferrari, S., Jacob, A., Cesana, D., Laugel, M., Beretta, S., Varesi, A., Unali, G., Conti, A., Canarutto, D., Albano, L., et al. (2022) · Cell stem cell · doi:10.1016/j.stem.2022.09.001 · Cited by 133
Tran, N. T., Danner, E., Li, X., Graf, R., Lebedin, M., de la Rosa, K., Kühn, R., Rajewsky, K., & Chu, V. T. (2022) · Science Advances · doi:10.1126/sciadv.abm9106 · Cited by 49
Ex vivo modification of hematopoietic stem and progenitor cells for gene therapy
Williams, D. A., Kohn, D. B., & Thrasher, A. J. (2025) · Molecular Therapy · doi:10.1016/j.ymthe.2025.03.058 · Cited by 11
Self-citation
Metadata
- Authors
- Kuo, C. Y., Long, J. D., Campo-Fernandez, B., de Oliveira, S., Cooper, A. R., Romero, Z., Hoban, M. D., Joglekar, A. V., Lill, G. R., Kaufman, M. L., Fitz-Gibbon, S., Wang, X., Hollis, R. P., & Kohn, D. B.
- ORCID
- Kuo, Caroline Y.
- Venue
- Cell Reports
- Publisher
- Elsevier BV
- Published
- 2018-05
- Volume
- 23
- Issue
- 9
- Pages
- 2606-2616
- ISSN
- 2211-1247
- PMC
- PMC6181643
- Cited by
- 139
- References
- 8
- Language
- en
- Licence
- Funders
- University of California, Los Angeles (K12HD34610-20, K12HD34610-19)
- California Institute for Regenerative Medicine (DISC2-10124)
- Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research, University of California Los Angeles
- Australasian Society of Clinical Immunology and Allergy
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- Kuo, C. Y., Long, J. D., Campo-Fernandez, B., de Oliveira, S., Cooper, A. R., Romero, Z., Hoban, M. D., Joglekar, A. V., Lill, G. R., Kaufman, M. L., Fitz-Gibbon, S., Wang, X., Hollis, R. P., & Kohn, D. B. (2018). Site-Specific Gene Editing of Human Hematopoietic Stem Cells for X-Linked Hyper-IgM Syndrome. Cell Reports. https://doi.org/10.1016/j.celrep.2018.04.103