Gene breakthrough after sad setback
Posted: November 19, 2012 at 2:40 pm
Marie McCullough, Inquirer Staff Writer Posted: Monday, November 19, 2012, 3:01 AM
The first gene therapy in the Western Hemisphere will soon be available in Europe.
The approval of Glybera by European regulators happened with little fanfare in September, in contrast to the hype that surrounded gene therapy 20 years ago.
But it is truly a breakthrough for the field, for people with the ultrarare disease it treats - and for University of Pennsylvania scientist James M. Wilson, a creator of the modified virus that delivers the therapeutic gene.
Wilson began to focus on finding safe, efficient gene-delivery viruses, or vectors, after a study he led caused the 1999 death of Arizona teenager Jesse Gelsinger - the world's first gene-therapy victim.
The vector in that study triggered a fatal immune system overreaction. The fallout set off years of government investigations and efforts to improve human-subject protections.
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Gene breakthrough after sad setback
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