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Correcting inborn errors of immunity: From viral mediated gene addition to gene editing

28 Feb 2023

Abstract excerpt

Allogeneic hematopoietic stem cell transplantation is an effective treatment to cure inborn errors of immunity. Remarkable progress has been achieved thanks to the development and optimization of effective combination of advanced conditioning regimens and use of immunoablative/suppressive agents preventing rejection as well as graft versus host disease. Despite these tremendous advances, autologous hematopoietic...

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PMID: 36863140
DOI: 10.1016/j.smim.2023.101731

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