Article
Targeted correction of a defective selectable marker gene in human epithelial cells by small DNA fragments.
Molecular therapy : the journal of the American Society of Gene Therapy - 1 Feb 2001
Colosimo A, Goncz K K, Novelli G, Dallapiccola B, Gruenert D C
Abstract excerpt
A novel gene targeting strategy, small fragment homologous replacement (SFHR), has been used to correct specific genomic lesions in human epithelial cells. The frequency of targeting was estimated to be 1-10%. However, given the genomic target, the cystic fibrosis transmembrane conductance regulator (CFTR) gene, it is difficult to accurately quantify targeting frequency. As an alternative to targeting CFTR,...
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