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	Provedor de dados BJMBR (1) Genet. Mol. Biol. (1) Autor Coura,Renata dos Santos (1) Martini,S.V. (1) Morales,M.M. (1) Nardi,Nance Beyer (1) Rocco,P.R.M. (1) Palavra-chave Adeno-associated virus (2) Gene therapy (2) AAV-based recombinant vectors (1) Cystic fibrosis (1) Cystic fibrosis transmembrane conductance regulator (1) Vectors (1) Mais... Tipo do documento Info:eu-repo/semantics/article (2) Ano 2011 (1) 2008 (1) País Brazil (2) Idioma Inglês (2)		Ordenar por:  Relevância Autor Título Ano Registros recuperados: 2 Primeira ... 1 ... Última A role for adeno-associated viral vectors in gene therapy Genet. Mol. Biol. Coura,Renata dos Santos; Nardi,Nance Beyer. Gene therapy constitutes a therapeutic intervention based on modification of the genetic material of living cells, by correcting genetic defects or overexpressing therapeutic proteins. The success of gene therapy protocols depends on the availability of therapeutically suitable genes, appropriate gene delivery systems and proof of safety and efficacy. Recent advances on the development of gene delivery systems, particularly on viral vectors engineering and improved gene regulatory systems, have led to marked progress in this field. Although the available vector systems can successfully transfer genes into cells, the ideal delivery vehicle has not been found. In this context, adeno-associated virus vectors (AAV) are arising as a promising tool for a wide... Tipo: Info:eu-repo/semantics/article Palavras-chave: Adeno-associated virus; AAV-based recombinant vectors; Gene therapy. Ano: 2008 URL: http://www.scielo.br/scielo.php?script=sci_arttext&pid=S1415-47572008000100001 Adeno-associated virus for cystic fibrosis gene therapy BJMBR Martini,S.V.; Rocco,P.R.M.; Morales,M.M.. Gene therapy is an alternative treatment for genetic lung disease, especially monogenic disorders such as cystic fibrosis. Cystic fibrosis is a severe autosomal recessive disease affecting one in 2500 live births in the white population, caused by mutation of the cystic fibrosis transmembrane conductance regulator (CFTR). The disease is classically characterized by pancreatic enzyme insufficiency, an increased concentration of chloride in sweat, and varying severity of chronic obstructive lung disease. Currently, the greatest challenge for gene therapy is finding an ideal vector to deliver the transgene (CFTR) to the affected organ (lung). Adeno-associated virus is the most promising viral vector system for the treatment of respiratory disease because it... Tipo: Info:eu-repo/semantics/article Palavras-chave: Gene therapy; Adeno-associated virus; Cystic fibrosis; Cystic fibrosis transmembrane conductance regulator; Vectors. Ano: 2011 URL: http://www.scielo.br/scielo.php?script=sci_arttext&pid=S0100-879X2011001100004 Registros recuperados: 2 Primeira ... 1 ... Última

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