First successful CRISPR gene-editing directly in the blood of a live patient

Originally published at: First successful CRISPR gene-editing directly in the blood of a live patient | Boing Boing


This could be very interesting for genetic diseases, definitely one of those moments where a medical science opens up a world of new treatments…


In the absence of special sea slugs, human ingenuity has found a way to do the splicing.


shh don’t tell the MAGA crowd that scientists are working on stuff


Great. Now, can you repair my genetics to remove my Neurofibro Mitosis?

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I feel like the first time this goes wrong someone is going to die a horrible death…

Just how did Dr. Crusher’s nano-bots do this work?

Please insert Ian Malcolm clip of “just because you can, doesn’t mean you should.”

Oh the fundies and bible thumpers of all sorts of religions are going to absolutely librarian-pooh.

Tampering with god’s plans and all that. Well, if this god doesn’t want his stuff tampered with, put a write protect on it so there’s no way to do things like this.

Should be easy enough for a being who can simply wish a universe into existence and has actual warehouses full of snow and wind and hail.
“Job 38:22”

Those of us with genetic issues that this might, in time, fix, can’t see it arrive fast enough.

Meanwhile, I’ll go watch Wrath of Khan for the heck of it,. :slight_smile:

Since this goes through the whole bloodstream I’m curious if this method also updates the genetic code of the germ cells. In earlier efforts at gene therapy researchers typically took extra care to NOT deliver any new instructions to germ cells, just to be certain that they wouldn’t pass on any engineered genes to future generations in case something went wrong or there were issues they didn’t account for.

Once we do get really good at genetic engineering and have a well established track record then passing on the new genes to offspring would be a good thing, as families with histories of bad genetic diseases could finally end it.

In the case of people who are past reproductive age it’s not really an issue, although men have been known to father children pretty late in life…


The NPR story doesn’t give much detail, but a target-specific guide RNA sequence was likely used. That allows the crispr/cas9 cassette to only transfect certain cell types which express the chosen RNA sequence, in this case the target would be limited to liver cells.

In research it has reached the point that single cell RNA sequencing can be used alongside crispr to create specific cassettes that will only transfect one very narrowly defined cell type.


I do worry about unintended consequences of this kind of gene alteration. Case in point, the CRISPR twins, where the scientist altered their genes to make them immune to HIV, but experts say it will likely have the side effect of making them smarter, but who knows.

But as the saying goes, you can’t put the genie back in the bottle. I believe this does mark a singularity in the long-term biological evolution of our species, as well as the planet (and beyond?). We now have the ability to modify our own biology, and create our own future generations, as well as that of other life on the planet. For good or evil. The fears of science fiction stories will soon be real.

Maybe I should have read the article better, but does the patient need to repeat this therapy?

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