Gene Therapy

Study data from Central South University update understanding of gene therapy

Published in Hospital Business Week, May 25th, 2008

"Vector systems to deliver, integrate and express therapeutic genes in host cells are essential for gene therapy. In the present study, we investigated a novel vector system for integration and expression of a transgene," scientists in Changsha, People's Republic of China report.

"In this system, the transgene expression was driven by an endogenous RNA polymerase I (Pot I) promoter after being integrated into the ribosomal DNA (rDNA) locus. Human coagulation factor IX coding sequence (FIX), with an internal ribosome entry sites element at its leader region, was targeted into the 18S rDNA locus via homologous recombination. FIX protein expression, which was under the...

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Source: Hospital Business Week (2008-05-25)

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