Jim Wilson offers a safer solution to some of the industry's gene therapy woes
Jim Wilson has been warning for a while about the dangers of the viral vectors used in most gene therapies. Two years ago he walked away from Solid Biosciences as he published a paper showing the potential adverse neurological effects of AAV vectors. Last year, he called the field’s progress on vectors “embarrassing.”
Now the gene therapy pioneer, whose tragic 1999 trial showed the deadly risks of the first generation of vectors, is offering a solution. On Wednesday, he and a team of researchers at the University of Pennsylvania published a paper in Science Translational Medicine showing a way of modifying the vectors so they correct genes without hitting a delicate clusters of neurons that, when disturbed, can cause dangerous side effects.
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