Novel Method Of Gene Replacement Reported By University Of Washington R...( rgeted cells. The highest rates occurre...)

"This level of exchange is perhaps 10,000 to 100,000 times better than has previously been achieved in normal human cells," said Russell. "There are some diseases such as hemophilia that could perhaps be cured with only modest improvements in the gene correction rate. Many diseases could be cured if future research enables us to correct genes in 10 percent of the targeted cells."

As a vector, the researchers used a virus called adeno-associated virus 2 (AAV), a single-stranded DNA virus capable of integrating into the chromosomes of mammals. Until this research, the potential of AAV vectors for gene targeting had not been explored. Russell and Hirata found that such vectors can target chromosomal genes at high frequencies and introduce modifications without creating additional mutations.

In an accompanying News & Views article, Dr. Alan Bernstein of the Samuel Lunenfeld Research Institute in Toronto states that the UW researchers' achievement is an important step toward efficient gene therapy, and that their results, in combination with similar advances on other fronts, suggest that the technical obstacles to efficient, targeted replacement of defective genes will eventually be overcome.

The research was supported by grants from the American Society of Hematology, the Lucile P. Markey Charitable Trust, the March of Dimes Birth Defects Foundation and the National Institutes of Health.

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Contact: Dr. David W. Russell
drussell@u.washington.edu
(206) 616-4562
University of Washington
30-Mar-1998