Adeno-associated virus for cystic fibrosis gene therapy
Braz. j. med. biol. res
; 44(11): 1097-1104, Nov. 2011. ilus
Artigo
em Inglês
| LILACS
| ID: lil-604280
Biblioteca responsável:
BR1.1
ABSTRACT
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.
Texto completo:
Disponível
Coleções:
Bases de dados internacionais
Base de dados:
LILACS
Assunto principal:
Terapia Genética
/
Adenovírus Humanos
/
Regulador de Condutância Transmembrana em Fibrose Cística
/
Fibrose Cística
/
Vetores Genéticos
Tipo de estudo:
Fatores de risco
Limite:
Humanos
Idioma:
Inglês
Revista:
Braz. j. med. biol. res
Assunto da revista:
Biologia
/
Medicina
Ano de publicação:
2011
Tipo de documento:
Artigo
País de afiliação:
Brasil
Instituição/País de afiliação:
Universidade Federal do Rio de Janeiro/BR