Scientists Edited Genes in a Living Person and Saved His Life

Note: from May 2025. https://gwern.net/doc/genetics/editing/2025-musunuru.pdf

Thanks! We've put this link in the header of the thread.

Well, CRISPR-Cas9 as a tool for genetic engineering was only invented in 2012. A 13-year translation timeline is not unusual, maybe even unusually fast. CAR-T cell therapy for cancer took 30 years from discovery to clinic. It took about 30 years to go from the early attempts to use engineered lipid nano particles for drug delivery to the first FDA-approved medication using them, doxil.

With CRISPR, it took a long time to figure out how to reliably edit just the gene you want and acceptably minimize off-target edits, including by delivering the therapeutic to just the organ affected and getting the dose and release right.

The public is understandably leery about experimental medical techniques. If they had killed this newborn child with CRISPR therapy, then it might have set created a backlash delaying translation of this technique for years, possibly decades.

In biomedicine, we’re always looking for therapies that approximate the level of precision control available in software. Unfortunately, it’s never more than an approximation, and our ability to measure and predict the size of that error is always limited. That is why the field moves slowly.

Wow this is amazing. Definitely the best biology video I've seen.

I think he actually genetically engineered his gut bacteria rather than any of his own cells there, right?

I thought (maybe mistakenly) that your body developed immunity to the adenovirus used to edit your own DNA, and so this trick could only be used once? Am I wrong?

With Trump it appears a window is closing on the development of important technologies and research. I doubt we will enter a new dark age, but in some areas, progress is likely to slow, which in turn will be used as evidence that it isn't worth funding, which will cause funding cuts and result in even slower progress. Everyone is racing to get stuff done, because there might not be the money tomorrow. It seems likely to me that longer term, more ambitious projects are probably being sidelined because there isn't time to complete them before the pencil pushers in the whitehouse defund them.

Look at how the Nixon administration and congress gutted NASA. It took nearly 40 years to crawl out of the hole in the roadmaps that their shortsighted stupidity created. We could have had reusable rockets, aerospike engines and Nuclear Thermal Propulsion in the early 1980s. Instead we got halfway measures like the space shuttle and the ISS that both ate budget but didn't create the required innovation for lower cost to orbit.

Unfortunately one of the common responses is "cancer is still deadly doesn't seem like they're doing anything". These therapies aren't widely known and people are willing to throw them out for that reason

I updated to version 10.5.7 and now I see Samsung ads in my peripheral vision, wtf

the good old days when updates actually improved product quality :)

If the affected protein is actively causing problems, then you'd need to get most of the cells. But if it's just inert and not doing its job then you only need to get enough cells to get the production of the right version up to a somewhat functional level.

It's like trying to persuade enough people (cell) to change their mind so a social revolution takes place (cure). The argument (gene vector) must be very popular.

> Don't you have to modify every cell (or affected type of cell) with a new copy of the DNA?

It depends on the specific disorder that’s being treated. For some of them, targeting a fraction of the affected cells is enough to get the desired effect.