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Efficiency of gene transfer for restoration of normal airway epithelial function in cystic fibrosis.
Johnson, L G; Olsen, J C; Sarkadi, B; Moore, K L; Swanstrom, R; Boucher, R C.
Afiliação
  • Johnson LG; Department of Medicine, University of North Carolina, Chapel Hill 27599-7020.
Nat Genet ; 2(1): 21-5, 1992 Sep.
Article em En | MEDLINE | ID: mdl-1284642
An important issue for in vivo gene therapy for cystic fibrosis (CF) is the percentage of cells within the CF airway that will require correction. In this study, we mixed populations of a CF airway cell line expressing either the normal cystic fibrosis transmembrane conductance regulator (CFTR) cDNA (corrected cells) or a reporter gene in defined percentages. As few as 6-10% corrected cells within an epithelial sheet generated C1-transport properties similar to sheets comprised of 100% corrected cells. Cell-cell coupling may serve as the mechanism for amplification of the functional effects of corrected cells. These data suggest that in vivo correction of all CF airway cells may not be mandatory.
Assuntos
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Base de dados: MEDLINE Assunto principal: Transfecção / Terapia Genética / Fibrose Cística Limite: Humans Idioma: En Revista: Nat Genet Assunto da revista: GENETICA MEDICA Ano de publicação: 1992 Tipo de documento: Article
Buscar no Google
Base de dados: MEDLINE Assunto principal: Transfecção / Terapia Genética / Fibrose Cística Limite: Humans Idioma: En Revista: Nat Genet Assunto da revista: GENETICA MEDICA Ano de publicação: 1992 Tipo de documento: Article