80beats – George Church, the geneticist behind the Personal Genome Project, is envisioning a package deal: get your genome sequenced, and he and his collaborators will develop a line of induced pluripotent stem cells (IPS) from your tissue, so in the future, you’ll be able upgrade your system with organs and tissues bearing both your genes and special extras like genes from centenarians. It’s combining stem cells with gene therapy.
Technology Review – When David Ewing Duncan asked George Church what he was most excited about right now, he answered without hesitation: “I’m thinking a lot about using regeneration as the key to treatments and keeping people healthy.”
TR: You mean regeneration using stem cells?
Church: Yes, induced pluripotent stem (IPS) cells (see, “Growing Heart Cells Just for You”). This is where I’m putting almost all of my chips these days, because it combines many of my interests–genomics, sequencing, epigenetics, synthetic biology, stem cells. I don’t think people have fully appreciated how quickly adult stem cells and sequencing and synthetic biology have progressed. They have progressed by orders of magnitude since we got IPS. Before that, they basically weren’t working.
An Entire Mouse has been made from IPS cells
Does IPS really work to accomplish this regeneration?
We have good evidence that you can create an entire mouse from IPS cells.
Has this been done?
This has been done (2009 Nature). They have used IPS cells to grow a mouse, and they made IPS cells from that mouse. They’re totipotent [able to make an entire organism], not merely pluripotent. We haven’t done this for humans for obvious ethical reasons, but we will do it. As far as I know the mice have done fine.
We have a recent paper in Nature that shows that when you make human induced pluripotent stem cells you actually do get mutations in coding regions at a slightly elevated level. But I think this is temporary. We’re going to use this information as an assay to make the process work better, to correct problems. You will be able to use this to improve the quality of gene therapy because that’s been the problem with gene therapy the last ten years.
Almost everything I’ve described has been done in rodents, so we’re talking about years, not decades. It’s shorter than the Human Genome Project [which took 13 years], not less expensive, but definitely shorter.
There is much to be worked out. But here’s the leap. If you want to accelerate this, you have to pick an intermediate target that doesn’t sound so scary. So you’ll start out with bone marrow patients. And you’re going to basically make a synthetic version of that patient’s bone marrow using IPS, which is going to work much better than the diseased bone marrow. And once this works that’s going to catch on like wildfire. And then you’ll do skin, and then you’ll do every other stem cell you can get.