Baby is healed with first personalized gene-editing treatment

>KJ has made medical history. The baby, now 9 ½ months old, became the first patient of any age to have a custom gene-editing treatment, according to his doctors.

This is _not_ the first human to be treated with a treatment under the wide umbrella of gene therapy based on their own edited genes. There probably is a more narrow first here but the technical details get lost in journalism which is a shame.

"Custom" in that this therapy was designed AFTER a specific patient showed a need, and then given to _that_ patient. In most every other context a particular class of disease is known, a drug designed, and then patients sought that have that disease that matches the purpose of the drug.

What's intriguing is not the 'custom' part, but the speed part (which permits it to be custom). Part of what makes CRISPR so powerful is that it can easily be 'adjusted' to work on different sequences based on a quick (DNA) string change - a day or two. Prior custom protein engineering would take minimum of months at full speed to 'adjust'.

That ease of manipulating DNA strings to enable rapid turnaround is similar to the difference between old-school protein based vaccines and the mRNA based vaccines. When you're manipulating 'source code' nucleic acid sequences you can move very quickly compared to manipulating the 'compiled' protein.

Okay, I'll bite: Who then was the first patient of any age to have a custom gene-editing treatment?

This is an AI-generated response, and is inaccurate.

That was one of the first cases of _germline_ gene editing using CRISPR - NOT "the first instance of gene editing." There have been quite a few other genetic editing tools that predate CRISPR, and there have been other edits using CRISPR that were not of the entire human's genome.

Ah yes, the "neutral" tone of AI generated content. "significant controversy and ethical concerns," sure.

That is not accurate, attempts at gene therapy in humans go back further than that.

https://www.whatisbiotechnology.org/index.php/science/summar...

There are always those concerns.

I want to say, maybe it's better to say first human under proper IRB/regulatory compliance. Some rogue academic in China tried it a few years ago, if I recall, but with absolutely no oversight and he was pilloried. Also I don't think there is much details about what he actually did...

https://www.npr.org/2023/06/08/1178695152/china-scientist-he...

What the Chinese guy (He) did was completely different. He permanently altered the germline in embryos, which means that every cell in the resulting baby is transformed permanently with the change he made. The work he did violated a wide range of good practice (specifically, the change he made didn't actually work for the goal he desired, and he also ignored all the ethical advice around this experiment, and avoided getting the necessary approvals).

This research is instead a therapy used to treat an already born baby, and it doesn't modify all the cells in the body. Many cells in the body that are transformed by this technique will eventually die and be replaced by clones of stem cells which weren't transformed. I haven't read in detail about whether this therapy targets stem cells, and how long term effective the treatment will be- hepatocytes (liver cells) turn over constantly, so I would expect if the treatment did not affect the hepatocyte stem cells, it would only last ~months and the treatment would have to be repeated.

The major difference is that was a hereditary change. So those changes could now diffuse throughout the species over time. As I recall it was a change that reduces vulnerability to HIV infection.

I know of at least one YouTuber who took a homemade treatment that alerted his GI system DNA to produce lactase so he could eat pizza again:

https://youtu.be/J3FcbFqSoQY

Do new liver cells always come from stem cells, not from dividing liver cells? Are those heppatocyte stem cells reside in the liver, or do they travel their via migration,.or blood, etc?

A quick search suggests that liver regen involves dividing mature liver cells to replace turnover. If so, I'd suspect that they'd continue to carry the.crispr edit forward.

I remember six kids treated with a HIV retrovirus based therapy in 2013 in Italy

That's not gene editing, but you could call it a gene therapy - he's introducing new fragments of DNA/RNA into his cells which then just float around and cause the right enzyme get made. Sometimes called upregulation. This is different to actually editing the existing DNA in the cell.

Good point - to be honest I didn’t realize the difference which is very significant from a treatment perspective.