Data/hora: 02/06/2024 10:33:59

Provedor de dados: | 56
País: | Brazil
Título: | Adeno-associated virus for cystic fibrosis gene therapy
Autores: | Martini,S.V.; Rocco,P.R.M.; Morales,M.M.
Data: | 2011-11-01
Ano: | 2011
Palavras-chave: | Gene therapy; Adeno-associated virus; Cystic fibrosis; Cystic fibrosis transmembrane conductance regulator; Vectors.
Resumo: | 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 has natural tropism for airway epithelial cells and does not cause any human disease. This review focuses on the basic properties of adeno-associated virus and its use as a vector for cystic fibrosis gene therapy.
Tipo: | Info:eu-repo/semantics/article
Idioma: | Inglês
Identificador: | http://www.scielo.br/scielo.php?script=sci_arttext&pid=S0100-879X2011001100004
Editor: | Associação Brasileira de Divulgação Científica
Relação: | 10.1590/S0100-879X2011007500123
Formato: | text/html
Fonte: | Brazilian Journal of Medical and Biological Research  v.44 n.11 2011
Fonte: | info:eu-repo/semantics/openAccess