Mount Sinai School of Medicine researchers cured PKU in mice with new gene therapy technique
Mount Sinai School of Medicine researchers developed a technique for inserting genes into specific sites on the genome in liver cells. The genes are inserted into non-coding regions of the genome so there is no danger of interfering with the functioning of other genes. Once inserted, the gene remains a permanent part of the cells genome. In a study published in this weeks PNAS, the researchers used this technique to cure phenylketonuria (PKU) in mice.
"To date gene therapy has relied upon vectors that randomly insert genes into the cells genome," explains Savio L. C. Woo, PhD, Professor and Chairman of Gene and Cell Medicine at Mount Sinai School of Medicine and corresponding author on the study. "The technique we developed identifies a specific sequence which only occurs in a few places in the mammalian genome. These sequences occur between genes so there is no danger of the insertion of the gene damaging existing genes in the cell.
Mount Sinai Press Office | EurekAlert!
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