Erythropoietin gene therapy leads to autoimmune anemia in macaques

被引：106

作者：

Gao, GP

Lebherz, C

Weiner, DJ

Grant, R

Calcedo, R

McCullough, B

Bagg, A

Zhang, Y

Wilson, JM

机构：

[1] Univ Penn, Dept Med, Div Med Genet, Gene Therapy Program, Philadelphia, PA 19104 USA

[2] Univ Penn, Dept Pathol & Lab Med, Philadelphia, PA 19104 USA

[3] Univ Penn, Div Hematol Oncol, Philadelphia, PA 19104 USA

[4] Childrens Hosp Philadelphia, Div Pulm Med, Philadelphia, PA 19104 USA

[5] Wistar Inst Anat & Biol, Philadelphia, PA 19104 USA

来源：

BLOOD | 2004年 / 103卷 / 09期

关键词：

D O I：

10.1182/blood-2003-11-3852

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

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.

引用

页码：3300 / 3302

页数：3

共 9 条

[1] Improvement of erythropoiesis in β-thalassemic mice by continuous erythropoietin delivery from muscle [J].