Combating oncogene activation associated with retrovirus-mediated gene therapy of X-linked severe combined immunodeficiency
Braz. j. med. biol. res
;
40(5): 601-613, May 2007. ilus, tab
Artículo
en Inglés
| LILACS
| ID: lil-449078
ABSTRACT
A successful gene therapy clinical trial that also encountered serious adverse effects has sparked extensive study and debate about the future directions for retrovirus-mediated interventions. Treatment of X-linked severe combined immunodeficiency with an oncoretrovirus harboring a normal copy of the gc gene was applied in two clinical trials, essentially curing 13 of 16 infants, restoring a normal immune system without the need for additional immune-related therapies. Approximately 3 years after their gene therapy, tragically, 3 of these children, all from the same trial, developed leukemia as a result of this experimental treatment. The current understanding of the mechanism behind this leukemogenesis involves three critical and cooperating factors, i.e., viral integration, oncogene activation, and the function of the therapeutic gene. In this review, we will explore the causes of this unwanted event and some of the possibilities for reducing the risk of its reoccurrence.
Texto completo:
Disponible
Índice:
LILACS (Américas)
Asunto principal:
Terapia Genética
/
Leucemia
/
Enfermedades por Inmunodeficiencia Combinada Ligada al Cromosoma X
Tipo de estudio:
Estudio de etiología
/
Factores de riesgo
Límite:
Humanos
Idioma:
Inglés
Revista:
Braz. j. med. biol. res
Asunto de la revista:
Biologia
/
Medicina
Año:
2007
Tipo del documento:
Artículo
/
Documento de proyecto
País de afiliación:
Brasil
Institución/País de afiliación:
Universidade de São Paulo/BR
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