Myelodysplasia and transgene inactivation in X-CGD-γ retroviral gene therapy: The usual suspects and new players

Affiliations

¹ Infection, Immunity, and Inflammation Teaching and Research Department, Great Ormond Street Institute of Child Health, University College London, London, UK.
² Infection, Immunity, and Inflammation Teaching and Research Department, Great Ormond Street Institute of Child Health, University College London, London, UK. Electronic address: g.santilli@ucl.ac.uk.

Thomas E Whittaker et al. Mol Ther. 2023.

. 2023 Dec 6;31(12):3367-3368.

doi: 10.1016/j.ymthe.2023.11.007. Epub 2023 Nov 18.

¹ Infection, Immunity, and Inflammation Teaching and Research Department, Great Ormond Street Institute of Child Health, University College London, London, UK.
² Infection, Immunity, and Inflammation Teaching and Research Department, Great Ormond Street Institute of Child Health, University College London, London, UK. Electronic address: g.santilli@ucl.ac.uk.

No abstract available

Conflict of interest statement

Declaration of interests The authors declare no competing interests.

1. Uchiyama T., Kawai T., Nakabayashi K., Nakazawa Y., Goto F., Okamura K., Nishimura T., Kato K., Watanabe N., Miura A., et al. Myelodysplasia after clonal hematopoiesis with APOBEC3-mediated CYBB inactivation in retroviral gene therapy for X-CGD. Mol. Ther. 2023;31:3424–3440. - PubMed
1. Braun C.J., Boztug K., Paruzynski A., Witzel M., Schwarzer A., Rothe M., Modlich U., Beier R., Göhring G., Steinemann D., et al. Gene therapy for Wiskott-Aldrich syndrome--long-term efficacy and genotoxicity. Sci. Transl. Med. 2014;6:227ra33. - PubMed
1. Stein S., Ott M.G., Schultze-Strasser S., Jauch A., Burwinkel B., Kinner A., Schmidt M., Krämer A., Schwäble J., Glimm H., et al. Genomic instability and myelodysplasia with monosomy 7 consequent to EVI1 activation after gene therapy for chronic granulomatous disease. Nat. Med. 2010;16:198–204. - PubMed
1. Hinai A.A., Valk P.J.M. Review: Aberrant EVI1 expression in acute myeloid leukaemia. Br. J. Haematol. 2016;172:870–878. - PubMed
1. Salter J.D., Bennett R.P., Smith H.C. The APOBEC Protein Family: United by Structure, Divergent in Function. Trends Biochem. Sci. 2016;41:578–594. - PMC - PubMed