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Gene therapy cures inherited liver disease in rats

immediately right after it is injected into the body.

"Fifteen years ago, the goal was to get some correction of the problem," he said. "Most effects were transient. Now with these vectors, long-term correction with no long-term toxicity is possible. We need to find a way to avoid short term toxicity."

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Others who participated in this research include: Drs. Gabriele Toietta, Viraj P. Mane, Milton J. Finegold, Philip Ng, and Arthur L. Beaudet of BCM and Drs. Wilma S. Norona and Antony F. McDonagh of the University of California, San Francisco.


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Source:Baylor College of Medicine


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