Importancia de la terapia génica en la enfermedad granulomatosa crónica / Gene Therapy in chronic granulomatous disease
Iatreia
; Iatreia;18(3): 308-319, sept. 2005. ilus, tab
Article
em Es
| LILACS
| ID: lil-422953
Biblioteca responsável:
CO56.1
ABSTRACT
Reactive oxygen species (ROS) production by phagocytes is an important mechanism to kill invading microorganisms. Neutrophils from individuals with chronic granulomatous disease (CGD) do not produce ROS, thereby rendering these individuals more susceptible to infection. CGD results from mutations in the genes encoding essential subunits of respiratory burst NADPH oxidase, the enzyme complex necessary for the production of these reactive molecules. The absence of phagocyte ROS results in recurrent fungal and bacterial infections and inflammatory granulomas, associated with significant morbidity and mortality. Currently, the curative treatment is the allogenic bone marrow transplant (BMT); nevertheless, this therapy has some disadvantages including the HLA incompatibility, the immunosupression due to the myeloablative conditions necessary for the transplant and the high risk to develop graft vs. host disease. As an alternative to BMT the ex vivo gene therapy in hematopoietic stem cells has been intensely studied. Although this option could be the most appropriate treatment, it can give rise to other kinds of adverse effects. The genetic features of CGD have made it a very attractive candidate to be cured with gene therapy. This review summarizes and discusses the current advances about gene therapy and its application to CGD.
Palavras-chave
Texto completo:
1
Índice:
LILACS
Assunto principal:
Terapia Genética
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NADPH Oxidases
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Vetores Genéticos
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Doença Granulomatosa Crônica
Tipo de estudo:
Prognostic_studies
Idioma:
Es
Revista:
Iatreia
Assunto da revista:
MEDICINA
Ano de publicação:
2005
Tipo de documento:
Article