Erythropoietin gene therapy leads to autoimmune anemia in macaques
Erythropoietin gene therapy leads to autoimmune anemia in macaques
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DOI：
10.1182/blood-2003-11-3852
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发表时间：
2004-05-01
期刊：
影响因子：
20.3
通讯作者：
Wilson, JM
中科院分区：
文献类型：
--
作者：
Gao, GP;Lebherz, C;Wilson, JM
基因疗法正在考虑用于递送治疗性蛋白质。我们评估了通过肌内施用腺相关病毒（AAV）载体将激素促红细胞生成素（EPO）递送到食蟹猴中。正如预期，动物出现超生理水平的EPO和红细胞增多症。然而，由于对内源性和转基因衍生的EPO的自身免疫应答，在一些动物中随后发生严重贫血。这是基因治疗导致灵长类动物无意中产生自身免疫的第一个例子。(C)2004年，美国血液学会。
Gene therapy is being considered for the delivery of therapeutic proteins. We evaluated the delivery of the hormone erythropoietin (EPO) into cynomolgus macaques through intramuscularly administered adeno-associated virus (AAV) vectors. As expected, the animals developed supra-physiologic levels of EPO and polycythemia. However, severe anemia ensued in some animals because of an autoimmune response to endogenous and transgene derived EPO. This is the first example of gene therapy leading to inadvertent autoimmunity in primates. (C) 2004 by The American Society of Hematology.