Just this past April, a report in the journal Cell attempted just that in
a mouse model of SCID. You might ask why are they testing a method
intended to avoid the immune system in animals that lack immune systems?
Bottom line, they wanted to see whether the gene therapy would work in
the simplest possible system before doing more complex experiments. So
they created a clone of their sick mouse, and harvested Embryonic Stem
cells from it. They then treated the cells IN A FLASK. This is crucial,
because there are other methods besides viruses we can use to put DNA in
cells once we have them in a flask. The only disadvantage these
non-viral methods have is low efficiency, but that problem goes away if
we can isolate the cured cells and allow them to multiply before using
them for treatment. So they have what they hope are "cured" ES cells
that can become any part of the mouse they need. And they needed bone
marrow, so they differentiated the cells into bone marrow and implanted
them into the mouse.
Slide 20