Erythropoietin gene therapy leads to autoimmune anemia in macaques-Reference-Cited by-同舟云学术

Erythropoietin gene therapy leads to autoimmune anemia in macaques

Published:2004-05-01 Issue:9 Volume:103 Page:3300-3302
ISSN:0006-4971
Container-title:Blood
language:en
Short-container-title:

Author:

Gao Guangping¹,Lebherz Corinna¹,Weiner Daniel J.¹,Grant Rebecca¹,Calcedo Roberto¹,McCullough Beth¹,Bagg Adam¹,Zhang Yi¹,Wilson James M.¹

Affiliation:

1. From the Gene Therapy Program, Division of Medical Genetics, Department of Medicine, University of Pennsylvania; Department of Pathology and Laboratory Medicine, University of Pennsylvania; Division of Hematology/Oncology, University of Pennsylvania, Philadelphia; Division of Pulmonary Medicine, Children's Hospital of Philadelphia; and the Wistar Institute, Philadelphia, PA.

Abstract

Abstract 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 supraphysiologic 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 auto-immunity in primates. (Blood. 2004;103: 3300-3302)

Publisher

American Society of Hematology

Subject

Cell Biology,Hematology,Immunology,Biochemistry

Link

http://ashpublications.org/blood/article-pdf/103/9/3300/1699049/zh800904003300.pdf

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