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TERAPIA GENICA

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A pesar de que os vírus representam o sistema mais eficiente, o desenvolvimento de vetores hibridos que combinem vantagens de vetores virais e não virais trarão novas perspectivas para o aumento da eficiência dos protocolos de terapia gênica
beyer9.jpg Autor:
Nance Beyer nardi,
Columnista Experto de SIIC

Institución:
Departamento de Genética Universidad Federal do Rio Grande do Sul (UFRGS) RS, Brasil

Artículos publicados por Nance Beyer nardi,
Coautores
Melissa Camassola*  Luisa Maria Braga** 
Bióloga, Mestranda junto ao Programa de Pós-Graduação em Genética e Biologia Molecular UFRGS.*
M Sc, Doutoranda junto ao Programa de Pós-Graduação em Genética e Biologia Molecular UFRGS.**
Recepción del artículo
20 de Abril, 2004 		Primera edición
8 de Octubre, 2004 		Segunda edición, ampliada y corregida
7 de Junio, 2021

TERAPIA GENICA

Resumen
A terapia gênica surgiu no meio médico e científico como uma abordagem molecular visando tratamentos e profilaxias para diversos tipos doenças, inclusive aquelas que não respondem a terapias convencionais. O princípio básico deste procedimento é a transferência de material genético para tecidos ou células alvo. A aplicação de protocolos de terapia gênica tem encontrado bem mais problemas que o esperado quando a idéia foi inicialmente estabelecida, há quase duas décadas. Uma baixa eficiência dos métodos de transferência gênica, bem como dificuldades na manutenção da expressão dos transgenes, têm sido as principais limitações. Três tecnologias de transferência gênica principais estão em desenvolvimento: o desenho de vetores sintéticos, o aperfeiçoamento de vetores virais, e protocolos para a transferência ex vivo principalmente para células tronco. Muitos métodos diferentes têm sido descritos para a transferência gênica para células tronco. Apesar de que os vírus representam o sistema mais eficiente, o desenvolvimento de vetores hibridos que combinem vantagens de vetores virais e não virais trarão novas perspectivas para o aumento da eficiência dos protocolos de terapia gênica. Vários ensaios clínicos estão em andamento e prometem grandes benefícios no futuro.

Palabras clave
Terapia gênica, vetores virais e não virais, transgene, MPS I, IDUA


Artículo completo
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Extensión:  +/-6.71 páginas impresas en papel A4
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GENE THERAPY

Abstract
Gene therapy was introduced in the scientific and medical areas as a molecular approach for the treatment and prevention of several kinds of diseases, including those which do not answer to conventional therapies. The basis of the procedure is the transfer of genetic material to target cells or tissues. The application of gene therapy protocols has faced many more problems than what was foreseen when the idea was first developed almost two decades ago. Low gene transfer effectiveness and the lack of long-term expression maintenance have been the main limitations. Three main gene transfer technologies are under development: design of synthetic vectors, improvement of virus-derived vectors, and protocols for the ex vivo gene transfer to (mainly stem) cells. Many different methods have been described to transfer genes to stem cells. Although retroviruses represent the most effective transfer system, the development of hybrid vectors which sum up viral and non-viral advantages will certainly bring new perspectives for the effectiveness of gene therapy protocols. Several clinical trials are now under experimentation and promise great benefits in a near future.

Key words
Gene therapy, viral and non-viral vectors, transgene, MPS I, IDUA

Clasificación en siicsalud
Artículos originales > Expertos de Iberoamérica >
página   www.siicsalud.com/des/expertocompleto.php/

Especialidades
Principal: Genética Humana
Relacionadas: Farmacología, Inmunología, Medicina Interna



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