Regulated GATA1 expression as a gene therapy for Diamond-Blackfan anemia
Voit RA., Liao X, Caulier A, Cohen B, Armant M, Antoszewski M, Lu HY, Fleming TJ, Kamal E, Wahlster L, Roche AM, Everett JK, Petrichenko A, Huang M, Clarke W, Myers KC, Forester C, Perez-Atayde A, Bushman FD, Pellin D, Shimamura A, William DA, Sankaran VG. (2024) Cell Stem Cell DOI: 10.1016/j.stem.2024.10.012.
[DOI]
Gene therapy for congenital marrow failure syndromes – no longer grasping at straws?
Voit RA., Corey SJ. (2023) Haematologica
[DOI]
MECOM deficiency: from bone marrow failure to impaired B-cell development
Voit RA., Sankaran VG. (2023) Journal of Clinical Immunology 43(6):1052-1066.
[DOI]
CD11c regulates neutrophil maturation
Hou, L., Voit, RA., Shibamura-Fujiogi, M., Koutsogiannaki, S., Li, Y., Chen, Y., Luo, H., Sankaran, V., & Yuki, K. (2023) Blood Advances
[DOI]
A genetic disorder reveals a hematopoietic stem cell regulatory network co-opted in leukemia
Voit, RA., Tao, L., Yu, F., Cato, L. D., Cohen, B., Fleming, T., Antoszewski, M., Liao, X., Fiorini, C., Nandakumar, S., Wahlster, L., Teichert, K., Regev, A., & Sankaran, V. (2022) Nature Immunology 24, 69–83.
[DOI]
Multiomics on our multitudes
Voit, RA., Sankaran VG. (2022) Nature Genetics 54(10):1449-1450.
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Pathogenic BCL11A variants provide insights into the mechanisms of human fetal hemoglobin silencing
Shen, Y., Li, R., Teichert, K., Montbleau, K. E., Verboon, J. M., Voit, RA., & Sankaran, V. (2021) PLoS Genetics 17(10), e1009835.
[DOI]
A unified model of human hemoglobin switching through single-cell genome editing
Shen, Y., Verboon, J. M., Zhang, Y., Liu, N., Kim, Y. J., Marglous, S., Nandakumar, S. K., Voit, RA., Fiorini, C., Ejaz, A., Basak, A., Orkin, S. H., Xu, J., & Sankaran, V. (2021) Nature Communications 12(1), 4991.
[DOI]
Familial thrombocytopenia due to a complex structural variant resulting in a WAC ANKRD26 fusion transcript
Wahlster, L., Verboon, J. M., Ludwig, L. S., Black, S. C., Luo, W., Garg, K., Voit, RA., Collins, R. L., Garimella, K., Costello, M., Chao, K. R., Goodrich, J. K., DiTroia, S. P., O'Donnell Luria, A., Talkowski, M. E., Michelson, A., Cantor, A., & Sankaran, V. (2021) J Experimental Medicine 218(6).
[DOI]
CD11c regulates hematopoietic stem and progenitor cells under stress
Hou, L., Voit, RA., Sankaran, V., Springer, T., & Yuki, K. (2020) Blood Advances 4(24), 6086–6097.
[DOI]
Pyruvate kinase deficiency in a newborn with extramedullary hematopoiesis in the skin
Voit, RA., & Grace, R. (2020) Blood 136(6), 770.
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Sowing the Seeds of Clonal Hematopoiesis
Liggett, L. A., Voit, RA., & Sankaran, V. (2020) Cell Stem Cell 27(2), 195–197.
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Stabilizing HIF to Ameliorate Anemia
Voit, RA., & Sankaran, V. (2020) Cell 180(1), 6.
[DOI]
Nuclease-mediated gene editing by homologous recombination of the human globin locus
Voit, RA., Hendel, A., Pruett-Miller, S. M., & Porteus, M. H. (2014) Nucleic Acids Research 42(2), 1365–1378.
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SAPTA: a new design tool for improving TALE nuclease activity
Lin, Y., Fine, E. J., Zheng, Z., Antico, C. J., Voit, RA., Porteus, M. H., Cradick, T. J., & Bao, G. (2014) Nucleic Acids Research 42(6), e47.
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An erythroid enhancer of BCL11A subject to genetic variation determines fetal hemoglobin level
Bauer, D., Kamran, S. C., Lessard, S., Xu, J., Fujiwara, Y., Lin, C., Shao, Z., Canver, M. C., Smith, E. C., Pinello, L., Sabo, P. J., Vierstra, J., Voit, RA., Yuan, G. C., Porteus, M. H., Stamatoyannopoulos, J. A., Lettre, G., & Orkin, S. H. (2013) Science 342(6155), 253–257.
[DOI]
Generation of an HIV resistant T-cell line by targeted “stacking” of restriction factors
Voit, RA., McMahon, M. A., Sawyer, S. L., & Porteus, M. H. (2013) Molecular Therapy 21(4), 786–795.
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