I've commented on this around here before. You read lots of junk about cancer "cures" in the popular press. They are almost always in mice or something and I or someone else with a similar background always feels compelled to weigh in and remind folks that it's a long way from curing lab rats to curing people.<p>This however, is the real deal. It's quite remarkable and there's likely more stories like this for other diseases on the way.<p>I went to a gene therapy session this fall at the American Society of Human Genetics conference in Boston and was blown away by some of the success people are having. I quipped to colleagues that I felt like I was in a science fiction movie. The most remarkable one was where they used an approach similar to the one here to cure a fatal metabolic disorder (relaying this from memory, so some of my recollection may be off). Kids with the disorder have a busted enzyme that causes slow degeneration of neurons. They don't live past 6 or 7 if I recall. The team showed how modifying a certain kind of stem cell found in the body normally to have the correct copy of the enzyme cured several patients. The corrected cells naturally move to the brain where they differentiate into glial cells and produce the correct copy of the enzyme. It turns out that because the neurons in the brain are starved for this enzyme, they express receptors that allow them to take it up from the environment. So the repaired glial cells supply enzyme to the entire brain (i.e. it's not necessary to modify every neuron in the brain to have a correct copy of the enzyme). They can completely cure kids with this approach. All of their muscular and neurological tests are 100% normal.<p>They had videos of these kids running around and playing just as if nothing was wrong. In one case, a younger brother lived but his older sister (who was too old when the therapy came out) had died. It was hard not to get choked up looking at their smiling, happy faces as they ran around, thinking that if this therapy hadn't existed, they would be in a nearly vegetative state.<p>Gene therapy had a rough start with the early setbacks, but I'm getting the sense that the tide is rapidly turing.