An essential for gene repair that fixes the problem without corrupting other genes (possibly causing cancer) is targeting the repair to the specific gene with the problem.
We’ve already seen the use of “zinc finger” nucleases to repair blood mutations in mice; now for the first time, scientists have used them to repair a defective gene in human stem cells: repairing a single nucleotide mutation without affecting anything else.
This work follows work in the same lab using different enzymes to achieve the same precision.
The combination of precision editing with pluripotent stem cells is the first step toward genetic repair of human organs (think diabetes, a host of metabolic diseases involving the liver, blood diseases…).
