Erythropoietin gene therapy leads to autoimmune anemia in macaques.
Blood
; 103(9): 3300-2, 2004 May 01.
Article
em En
| MEDLINE
| ID: mdl-14695227
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.
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Coleções:
01-internacional
Base de dados:
MEDLINE
Assunto principal:
Terapia Genética
/
Eritropoetina
/
Anemia Hemolítica Autoimune
Limite:
Animals
Idioma:
En
Revista:
Blood
Ano de publicação:
2004
Tipo de documento:
Article
País de afiliação:
Estados Unidos