New gene therapy fixes mistakes

Existing techniques only compensate for mutations

By Tina Hesman Saey, 14:18 PM June 27, 2011

A new type of gene therapy allows scientists to fix DNA defects directly. That’s a potentially revolutionary improvement on present gene therapy techniques, which introduce working genes to cells — but not into the genetic library itself.

“This is a major leap of the technology,” says John Rossi, a molecular geneticist at the Beckman Research Institute of City of Hope in Duarte, Calif.

Working with newborn mice, researchers led by Katherine High at the Children’s Hospital of Philadelphia ...

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