Gene Therapy Advances

Three interesting advances in gene therapy have recently been reported.

The first reports gold nano particles to deliver gene therapy directly to skin cells, simply by including them in commercial moisturising cream. This particular therapy uses siRNA to specifically target and silence disease-causing genes.

Even more exciting is the discovery of a DNA-cutting enzyme which is guided to its cutting site by RNA. Since RNA recognises DNA by easily worked out and synthesised base pairing (unlike the much more difficult to engineer direct recognition by the protein’s 3-dimensional structure), this has the potential for a universal gene targeting system.

Finally, other scientists have found that zinc finger nucleases can directly penetrate cellsso they don’t need to be delivered using DNA vectors: this could lead to much simpler and safer ways of editing genes in stem cells or differentiated cells. (For more on what’s good about zinc finger nucleases, see here.)

The rate the science is advancing, it can’t be too many years before routine gene therapy is a reality.