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首页> 外文期刊>Proceedings of the National Academy of Sciences of the United States of America >Correction of hemophilia as a proof of concept for treatment of monogenic diseases by fetal spleen transplantation
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Correction of hemophilia as a proof of concept for treatment of monogenic diseases by fetal spleen transplantation

机译：血友病的纠正作为胎儿脾移植治疗单基因疾病的概念证明

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摘要

Previous clinical attempts to correct genetic deficiencies such as hemophilia or Gaucher disease by transplantation of allogeneic spleen were associated with aggressive graft versus host disease, mediated by mature T cells derived from the donor spleen. We show that a fetal pig spleen harvested at the embryonic day 42 stage, before the appearance of T cells, exhibited optimal growth potential upon transplantation into SCID mice, and the growing tissue expressed factor VIII. Transplantation of embryonic day 42 spleen tissue into hemophilic SCID mice led to complete alleviation of hemophilia within 2-3 months after transplant, as demonstrated by tail bleeding and by assays for factor VIII blood levels. These results provide a proof of principle to the concept that transplantation of a fetal spleen, obtained from a developmental stage before the appearance of T cells, could provide a novel treatment modality for genetic deficiencies of an enzyme or a factor that can be replaced by the growing spleen tissue.
机译：先前的临床尝试通过同种异体脾脏移植来纠正遗传缺陷，例如血友病或高雪氏病，与由供体脾脏衍生的成熟T细胞介导的侵袭性移植物抗宿主病有关。我们表明，在胚胎第42天收获的胎猪脾，在T细胞出现之前，在移植到SCID小鼠中后表现出最佳的生长潜力，并且生长的组织表达了VIII因子。胚胎第42天的脾脏组织移植到血友病SCID小鼠中可导致移植后2-3个月内血友病的完全缓解，如尾巴出血和VIII因子血药浓度的测定所证明的。这些结果为以下概念提供了原理上的证明：从T细胞出现之前的发育阶段获得的胎儿脾脏的移植可以为酶或可由其替代的因子的遗传缺陷提供新的治疗方法。脾组织生长。

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