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Nonconditioned ADA-SCID gene therapy reveals ADA requirement in the hematopoietic system and clonal dominance of vector-marked clones.
Uchiyama T, Takahashi S, Nakabayashi K, Okamura K, Edasawa K, Yamada M, Watanabe N, Mochizuki E, Yasuda T, Miura A, Kato M, Tomizawa D, Otsu M, Ariga T, Onodera M. Uchiyama T, et al. Among authors: edasawa k. Mol Ther Methods Clin Dev. 2021 Oct 16;23:424-433. doi: 10.1016/j.omtm.2021.10.003. eCollection 2021 Dec 10. Mol Ther Methods Clin Dev. 2021. PMID: 34786435 Free PMC article.
Myelodysplasia after clonal hematopoiesis with APOBEC3-mediated CYBB inactivation in retroviral gene therapy for X-CGD.
Uchiyama T, Kawai T, Nakabayashi K, Nakazawa Y, Goto F, Okamura K, Nishimura T, Kato K, Watanabe N, Miura A, Yasuda T, Ando Y, Minegishi T, Edasawa K, Shimura M, Akiba Y, Sato-Otsubo A, Mizukami T, Kato M, Akashi K, Nunoi H, Onodera M. Uchiyama T, et al. Among authors: edasawa k. Mol Ther. 2023 Dec 6;31(12):3424-3440. doi: 10.1016/j.ymthe.2023.09.004. Epub 2023 Sep 13. Mol Ther. 2023. PMID: 37705244