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Baby is healed with first personalized gene-editing treatment

(www.nytimes.com)
1162 points jbredeche | 4 comments | 15 May 25 18:06 UTC | HN request time: 0.698s | source
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colechristensen ◴[15 May 25 19:25 UTC] No.43998409[source]▶
>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.

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1. caycep ◴[15 May 25 20:05 UTC] No.43998771[source]▶
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...

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2. dekhn ◴[15 May 25 20:25 UTC] No.43998960[source]▶
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.

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3. zardo ◴[15 May 25 20:35 UTC] No.43999061[source]▶
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.
4. SubiculumCode ◴[16 May 25 05:29 UTC] No.44002101[source]▶
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.