re: Human Gene Therapy

There are certain protocols involving stem cells in which the actual editing of your genomic data isn't done in vivo.

If you weren't aware, we can essentially take any cell in the body and induce the formation of patient specific stem cells, which are known as inducible stem cells. This is done by exposing these cells to four specific transcription factors which effectively reprogram your cells to a pluripotent state.

Once induced, the cells can be transfected with a viral cassette containing the therapeutic gene. If the gene is installed at the correct genomic coordinate, they can then be allowed to differentiate in vitro before being transplanted back into the donor.

This protocol was used a few years ago to effectively cure mice afflicted with sickle cell anemia.