Gene Therapy
Lengthened targeting arms do not increase HR efficiency in stem-cell beta-globin locus
Published in Gene Therapy Weekly, October 2nd, 2003
"The correction of mutant beta-globin genes has long been a therapeutic goal for patients with beta-thalassemia or hemoglobinopathies," scientists in the United States explained. "The use of homologous recombination (HR) to achieve this goal is an attractive approach because it eliminates the need to include regulatory, sequences in the therapeutic construct, and it eiliminates mutagenesis induced by random integration."
"However, HR is a very inefficient process for gene correction, and its efficiency is probably locus-dependent," according to Z.H. Lu and...
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Source: Gene Therapy Weekly (2003-10-02)
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