Long-term and real-world safety and efficacy of retroviral gene therapy for adenosine deaminase deficiency

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DOI:10.1038/s41591-023-02789-4
文献类型：期刊论文
补充材料：只需要正文
文献链接：https://doi.org/10.1038/s41591-023-02789-4

Nature

Abstract：Adenosine deaminase (ADA) deficiency leads to severe combined immunodeficiency (SCID). Previous clinical trials showed that autologous CD34+ cell gene therapy (GT) following busulfan reduced

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