this post was submitted on 17 Mar 2025
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[–] andros_rex@lemmy.world 9 points 12 hours ago (1 children)

By all account what he did worked.

Incorrect.

Two days after Lulu and Nana were born, their DNA were collected from blood samples of their umbilical cord and placenta. Whole genome sequencing confirmed the mutations.[124] However, available sources indicate that Lulu and Nana are carrying incomplete CCR5 mutations. Lulu carries a mutant CCR5 that has a 15-bp in-frame deletion only in one chromosome 3 (heterozygous allele) while the other chromosome 3 is normal; and Nana carries a homozygous mutant gene with a 4-bp deletion and a single base insertion.[125] He therefore failed to achieve the complete 32-bp deletion.[109] Moreover, Lulu has only heterozygous modification which is not known to prevent HIV infection.[120] Because the babies' mutations are different from the typical CCR5Δ32 mutation it is not clear whether or not they are prone to infection.[125] There are also concerns about adverse effect called off-target mutation in CRISPR/Cas9 editing and mosaicism, a condition in which many different cells develop in the same embryo.[124] Off-target mutation may cause health hazards, while mosaicism may create HIV susceptible cells.

[–] arrow74@lemm.ee 0 points 12 hours ago* (last edited 12 hours ago) (1 children)

Per the wikipedia page it states that it is not clear if it effective because they're not going to intentionally infect the children to test it. But we see the results specifically on the targeted gene. That's a success and demonstrates the technology works.

I'd argue the folly was inserting an artificial gene as opposed to the natural gene that we already know works. Either way the technology showed expression on the correct gene, that is a success.

We'd be having a better discourse on this if his results weren't banned from every journal and not studied.

[–] andros_rex@lemmy.world 6 points 12 hours ago (1 children)

Read that section I pasted in again.

“Lulu has only heterozygous modification which is not known to prevent HIV infection.”

It’s not the results are “banned from every journal” - it’s that doing ad hoc CRISPR experiments is not going to meet peer review. Doing random things because you want to see what happens is not how science works.

[–] arrow74@lemm.ee 1 points 12 hours ago (1 children)

Having a heterozygous deletion is still effecting the right gene. Without knowing both of her parents genetics it's hard to say if it was natural. What he did could produce either a heterozygous or homozygous result on the gene, but only the homozygous presentation is effective at prevention.

So 1 was a full success and the other showed activation on the appropriate gene, but not enough to confer resistance. Although it is possible it does since he used an artificial gene. We know the natural one is not effective in a heterozygous presentation. I still think that was his greatest mistake. He should have just used the naturally effective gene.

You do make a good point with the full backing rigor of the scientific method this procedure would always be successful.

[–] andros_rex@lemmy.world 4 points 12 hours ago* (last edited 12 hours ago) (2 children)

You do make a good point with the full backing rigor of the scientific method this procedure would always be successful.

What? Even highly effective treatments with ample research backing will not “always be successful.” (Not just in genetics. Across the board.)

Again, as the excerpt I copied in shows, there are also RISKS with CRISPR. Things like mosaicism, things like half of your cells having the modification and half not.

Do you have any background in biology? Can you explain why a gene that only conveys resistance in a homozygous genotype would be magically effective in a heterozygous because it was artificial?

Can you define the terms “homozygous” and “heterozygous” even?

[–] JacksonLamb@lemmy.world 2 points 8 hours ago

This topic is flushing out some concerning people.

[–] arrow74@lemm.ee 1 points 11 hours ago (1 children)

I didn't say it was magic. Part of the issue is we don't know what modifications he made in making his artificial version. I won't pretend like there aren't a lot of unknowns there. It could alter the effectiveness in numerous ways.

[–] andros_rex@lemmy.world 3 points 9 hours ago

Yes - exactly. He didn’t know what was going to happen. When you don’t know what is going to happen, you don’t play with lives.