November 29, 2013
DNA Editing Biotech Start-Up
Using a method called CRISPR/Cas a start-up called Editas is harnessing anti-viral proteins from bacteria to edit DNA. Read the whole thing.
A new startup, backed with $43 million in venture investments, aims to develop treatments that could cure inherited diseases with a one-time fix based on a new method of genome editing.
This is a big deal. As we learn more about the functional significance of more genetic variants we will be able to find lots of individual genetic variants we have that are slightly harmful (genetic load). We will be able to make better cell therapies and grow better replacement organs by taking some cells from our body and then editing out all the harmful mutations.
Genetic editing will speed up the rate of genetic research because researchers will be able to create cell lines in culture to investigate the effects of different combinations of genetic variants.
For therapeutic uses this technology will be developed first to make better stem cell therapies for those with genetic diseases. Gradually as we learn more about genetic mutations that cause smaller amounts of harm it will be used to make better stem cell therapies for larger fractions of the population. Since we all have lots of mildly harmful mutations (genetic load) our stem cell therapies for rejuvenation will be able to replace our aged cells with cells that are both young and better than our cells were than when we were young.
We can make ourselves better in lots of ways. Lactose intolerant? Get some youthful intestinal cells that will let you drink milk. Got some other food intolerance? Fix it with genetic programming. Have your tendons always injured easily? We'll know which genetic variants make better tendons and, for that matter, better joints and better lungs. Play sports you've always wanted to play. What I want: the Apo A-1 Milano mutation. There are probably lots of other good ones that haven't been discovered yet which exist only in thousands or tens of thousands of people.
Some practical uses of genetic editing beyond stem cells include livestock genetic engineering. For example, put disease-resistant genetic variants into cows and sheep from wild animals. Or how about genetically engineering sheep to grow fur more like alpaca fur?
At a much later step DNA editing to make offspring will become really popular once it becomes possible to do it safely. First get rid of the genetic load. Just getting rid of the genetic load will increase beauty and boost intelligence. Then do edits to make beautiful children. Perhaps throw in some edits for athletic ability. Golf nuts with lousy games will want to make their kids naturals at golf.
Looks like we are going to get the technology for DNA editing before we get the knowledge for the meaning of the vast majority of genetic variants. We will see genetic editing done to create superior livestock before it is done on humans. Also, we will see genetic editing done by dog and cat breeders to produce healthier and smarter pets.
These usages will legitimize genetic editing. If national governments try to ban its use to make better babies I expect to see a lot of medical tourism where prospective parents travel abroad to a country with little regulation on allowed types of genetic enhancement for reproduction.
Different levels of strictness on genetic enhanced offspring will tend to create larger differences in abilities between the more and less affluent. The people with enough money to travel abroad to spend a few months starting a pregnancy will have a competitive advantage over the poor and only middle class who have to live with the restrictions (and slow regulatory approval processes) who can't afford to pay for a trip abroad to get genetic enhancements for their babies.
We might see a lot of medical tourism to Hong Kong or Singapore to get highly technically competent genetic editing to produce better eggs, sperm, and embryos.
Randall Parker, 2013 November 29 02:08 PM
The article seems to say that this is aimed at adults, not eggs, sperm, and embryos.
The human DNA is so complex that we must ask the following question: can removing a gene that causes disease cause other problems? It is very possible that for many years this kind of treatment will be very unpredictable. Maybe it will first be used on patients who are already at high risk of a specific disease so that they have nothing to lose.
If you have a mutant gene cranking out a protein that's destroying the body, the results are complex -- to be sure -- but then entropy produces high complexity. Actually, a rather ironic fact about clinical trials for treatment of ultimately fatal genetic diseases is they tend to exclude people who are in the advanced stages of the disease due a variety of factors related to managing the experiment and interpreting its results.
Wolf-dog, if a defective version (allele) of a gene is causing illness, if we can snip that out and replace it with a normal version then that's not going to cause any problems as it is clearly the defective gene that is the problem. For example, everyone who has Huntington's disease has a version of the defective gene responsible. But improving people through genetic engineering rather than just preventing genetic disease is more difficult because, unlike suffers of Huntington's disease, there is no single gene allele that all mathimatical geniuses have or all great composers have. And so there's no single gene allele we can give to children to reliably produce Mozarts. All we seem to be able find is gene alleles that are correlated with particular traits but which don't appear to be sufficient in of themselves to result in that particular trait. Also, a gene allele that's correlated with something good could also be correlated with something bad, which obviously can complicate things. It seems that there is a lot of complex interaction going between the external environment and the internal enviroment which consists of the other genes that any one particular gene has to work with. This does not mean it is not possible to improve people through genetic engineering, it just means that it is quite a complex problem, and we may not be able to consistantly produce great mathematicians because random processes play a significant part in development. For example, consider the fingerprints of identical twins. While there will be a basic similarity, genetically identical twins still have different fingerprints as random chance determines how they finally develop. There is also an element of randomness in handedness and one member of a pair of identical twins can be right handed and the other can be left handed. Because there is a great deal of chaos involved in development we won't be able to get consistent results from genetic engineering alone, but we certainly can increase the chances of hitting a particular target, whatever that target may be.
Of course the initial uses will be treatment of diseases. That is way less controversial. A start-up should make no mention of offspring genetic engineering. So that is why the world needs bloggers: to explain the unmentionable.
It is inevitable that once the technology matures it will also be used for making better offspring.
I think the designer babies applications are a long ways off. Prospective parents to be are risk averse with regards to their kids. Sure, they'll do all kinds of PGD to ensure their kids are free of genetic defects. PGD is a risk averse technology in the sense that the embryos are not modified in any way. De novo genetic engineering is a completely different story. Prospective parents will not consider this kind of technology until they be rest assured that it is completely safe. This will take several decades.
Self-modification (e.g. genetic therapy for adults) is a different story. This, along with anti-aging therapies will drive medical tourism for the foreseeable future.