February 16, 2014
Genetic Editing Tech To Make Gene Therapies Workable
Check out this Technology Review piece on recent advances that have made it possible to make small changes in genetic sequences in cells. CRISPR, TALENs and other genetic editing technologies are making precise genetic editing possible.
Two decades ago gene therapy was supposed to be the next big thing. Didn't happen. Turned out to be much harder and more dangerous than was hoped. The technologies to do genetic editing are finally getting developed. I expect a big acceleration in the rate of progress in both gene therapy and cell therapy as a result.
Really cheap genetic sequencing is a complementary technology because it will allow us to identify all the harmful mutations (genetic load). We all have hundreds or thousands of slightly harmful mutations and some of us have very harmful mutations. Imagine some cells taken from your body, extensively edited to remove all the harmful mutations (including mutations that accumulate with age). Then the improved and rejuvenated cells could be injected at various places in the body to start doing repairs. I am looking forward to the day when that becomes possible.
Randall Parker, 2014 February 16 09:15 PM
I like the idea of using edits to remove a bunch of the genetic load from stem cells, and also insert markers and a "kill switch". Rejuvenate the body using the stem cells. If one line goes cancerous or it's just time to clean out the oldest, most exhausted and least-optimized lines, use the kill switch.
What we really need to compliment this is a good synthetic chromosome technology. That way we have full control over the context of the new gene, and just need to disable the old. Also allows for insertion of much more complex genes.
We're not even at the point where we know why much of the medicine that regularly gets placed inside people works. Working out which genes are not optimal within the environment of a particular person is going to be difficult. But, you know, progress is being made. But for a while we may have to settle for such mundane things such as "reduced risk of cancer" or "no more Parkinson's".
Brett, an artifical genome might be the best approach. Make it as resistant to stuff ups as possible then suck out the old one out of cells and shove it in or replace cells entirely. It would have to be personalized so cells it's in wouldn't start produceing membrane protiens that would get them rejected by the body. An artificial genome could be much smaller and this would make it easier to have say three or more copies in the nucleus for error checking. Just how that might work I don't know, but the new genome wouldn't necessarily have to use DNA. We might come up with something better. And maybe we could come up with something different to RNA mediation too and come up with a better cell all together.