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Terapia gênica Gene therapy

Nance Beyer Nardi 1 Leonardo Augusto Karam Teixeira 2 Eduardo Filipe Ávila da Silva 1

Abstract Gene therapy is a medical intervention that involves modifying the genetic material of living cells to fight disease. Genes influence virtually every human disease, either by encoding for abnormal proteins, which are directly responsible for the disease, or by causing a susceptibility to environmental agents which induce it. Gene therapy is still experimental, and is being studied in clinical trials for many different types of diseases. The development of safe and effective methods of implanting normal genes into the human cell is one of the most important technical issues in gene therapy. Although much effort has been directed in the last decade toward improvement of protocols in human gene therapy, and in spite of many considerable achievements in basic research, the therapeutic applications of gene transfer technology still remain mostly theoretical. The potential for gene therapy is huge and likely to impact on all aspects of medicine. Key words Viral and nonviral vectors, Gene therapy protocols, Gene transfer
1 Departamento de Genética, Universidade Federal do Rio Grande do Sul. Av. Bento Gonçalves 9.500. Caixa Postal 1.5053 91501-970 Porto Alegre RS. nardi@vortex.ufrgs.br 2 Pesquisa Básica, Divisão de Medicina Experimental, Instituto Nacional do Câncer, Rio de Janeiro.

Resumo Terapia gênica é um procedimento médico que envolve a