Five years ago I wrote for the GSA weblog, Read/write access to your genomes? Using the past to jump to the future. The basic idea is that whole-genome analysis will start to become “normal” in the 2010s, while “writing” to the genome (through CRISPR) will only start to hit the mainstream in the 2020s.

For humans, where it is going to start and become ubiquitous over time is in Mendelian diseases. Basically, those diseases characterized by a large effect mutation that genetic engineering can target. If you have a genetics education, you know that sickle cell anemia is one of those diseases. Often, it is a classic example of heterozygote advantage. Those with one copy of the mutation are more resistant to malaria, while those with two copies exhibit disease.

Last year a young woman received treatment to “fix” the mutation. NPR has been following her, and the results are good, 1st Patients To Get CRISPR Gene-Editing Treatment Continue To Thrive:

Gray and the two other sickle cell patients haven’t had any complications from their disease since getting the treatment, including any pain attacks or hospitalizations. Gray has also been able to wean off the powerful pain medications she’d needed most of her life.

Prior to the treatment, Gray experienced an average of seven such episodes every year. Similarly, the beta thalassemia patients haven’t needed the regular blood transfusions that had been required to keep them alive.

“It is a big deal because we we able to prove that we can edit human cells and we can infuse them safely into patients and it totally changed their life,” says Dr. Haydar Frangoul at the Sarah Cannon Research Institute in Nashville. Frangoul is Gray’s doctor and is helping run the study.

I am probably biased because of my professional focus, but this may be the biggest story of 2018, Chinese scientists are creating CRISPR babies:

According to Chinese medical documents posted online this month (here and here), a team at the Southern University of Science and Technology, in Shenzhen, has been recruiting couples in an effort to create the first gene-edited babies. They planned to eliminate a gene called CCR5 in order to render the offspring resistant to HIV, smallpox, and cholera.

We knew this was coming. Soon. But now we can confirm it. It confirms my assumption that gene editing in the human context is going to mostly focus on preventing disease in the near future. In a world of low fertility, every expectant parent prays (literally or metaphorically) for a healthy child. After the child is born they can think about other things like how tall they are going to be or how smart they are. But health, that is always the number one concern.

From what I know the United States still has the largest number of top-flight researchers in the basic and applied sciences. American scientific culture, for all its faults, is second to none. But for various reasons, I can’t see America trying to keep up with the Chinese when it comes to gene editing of humans. CRISPR technology will probably be applied to other things, such as in applied plant and animal sciences, in this country.

The future is here. We’re just along for the ride….

Addendum: As someone who has read S. M. Stirling’s Draka series, I am getting a really weird feeling right now about the trajectory that I see for the next generation….

Update: A computational biologist at Fudan University doesn’t believe in the results:

I think the story about “Chinese CRISPR babies” is false.

I also think that there is much less resistance to the idea in China than in Judeo-Christian societies.

So, whether this story is true or not in 2018, it will most likely be true (in some form) by 2028.

— Luis Pedro Coelho (@luispedrocoelho) November 26, 2018

So as of now China is producing more scientific publications than the United States of America. But there’s quality and there’s quantity. I think most people would still American science is more cutting edge than Chinese science. And for cultural reasons that may stay true for a while longer.

But there is one area where China seems to be forging ahead and likely will make advances earlier than the USA: genetics, and genetic engineering in particular. The Wall Street Journal has a long piece, China, Unhampered by Rules, Races Ahead in Gene-Editing Trials. It turns out that Chinese have been doing human trials since 2015. Meanwhile, in the USA the greenlight has still not been given (though it seems close).

Honestly how quickly the Chinese are moving in human trials is alarming. Then again, this is a country with the highest number of executions in the world (some of this is sheer size, but it’s higher per capita than the USA). So we should keep perspective. There are many worse things that the Chinese are doing in relation to human rights that moving too fast in trials with cancer patients.

In any case, this comment jumped out at me:

In traditional drug development, too, human-trial rules can differ among countries. But China’s foray into human Crispr trials has some Western scientists concerned about the unintended consequences of using the wholly new tool—such as harm to patients—which could set back the field for everyone.

Western scientists the Journal interviewed didn’t suggest America’s stringent requirements should be weakened. Instead, many advocate an international consensus on ethical issues around a science that makes fundamental changes to human DNA yet still isn’t completely understood.

As a descriptive matter, I am highly skeptical of the possibility that “international standards” is going to involve the Chinese adhering to Western standards. If a genuine international consensus is going to emerge there has to be a give and take, which means that the very high threshold set for safety in human trials in the West may not apply in China.

The media is writing breathless stories about the recent CRISPR “embryo-editing”, In Breakthrough, Scientists Edit a Dangerous Mutation From Genes in Human Embryos.

The paper is out in Nature, Correction of a pathogenic gene mutation in human embryos.

My major confusion is that this is normal science. The breakthrough was the discovery of the power of CRISPR-Cas9. Once the discovery was made there was a literally stampede to use the technique because its power and ease was so manifest. What’s happening now is that the technique is getting more powerful and effective. I think it would surprise people if it didn’t get better.

A major problem for economists in modeling productivity growth is that innovation is unpredictable. But in this case the big innovation has occurred. The next few decades are likely going to see progressive and continuous improvement in the technology. Where that will lead us? Unpredictable….