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Three people with inherited diseases successfully treated with CRISPR

newscientist.com

11–20 of 74 posts

Re: Three people with inherited diseases successfully treated with CRISPR

#11
post #9

But will the drug approval agencies allow genetically engineered therapies for anything disruptive? For example, they've been blocking the caries vaccine for decades.

> blocking the caries vaccine for decades Please give more details.

There are no details to give, there never was a successful caries vaccine it’s just always been stuck at being worked on.

Big dental isn’t blocking it we just don’t have a good enough model and yes there isn’t a sufficient financial incentive to develop it really either.

Caries isn’t a big problem these days, it’s not then major cause of tooth problems as people grow older, dental hygiene and more importantly fluoride in water pretty much solved it for those population that were affected.

There is no financial reason to block a caries vaccine as it’s not going to have any impact on the industry, cosmetic and corrective procedures would still be just as in demand and gum disease is by far a bigger factor for tooth loss in adult patients than caries.

Not to mention that quite a lot of the “vaccines” weren’t traditional vaccines but rather replacement therapies where lacto acid producing bacteria would be replaced with strains that cannot produce it but can outcompete the lactobacillus flora in your mouth, these treatments are let’s say problematic since we have had little to no experience in flora replacement therapies and can’t predict or model the outcomes well.

Re: Three people with inherited diseases successfully treated with CRISPR

#13

It's interesting that they did not just undo the inherited disease. I assumed that, especially with Sickle Cell where we have a good understanding of how it works, they would go into Chromosome 11 and put it back how it "should" be with CRISPR. But instead they apply a workaround, ensuring continued fetal haemoglobin production. The article does not mention whether that's because putting Chromosome 11 back with CRISP…

One reason may be that a general fix that produces good hemoglobin is much more efficient than attempting to fix the original problem. People can have a near-limitless number of mutations that lead to defective proteins, so creating fixes for all those "bugs" can be very expensive. A general fix that works around the "bug" is cheaper, consistant, and more scalable. Another way to look at it: Would a per-patient fix b…

That’s true even if the mutation is the same it’s hard to actually fix it, while reactivating or over-expressing a gene that already exists and works is much easier.

For this specific case it’s also better because lesser coverage might still produce sufficient results since fetal hemoglobin out competes adult hemoglobin.

I think in general CRISPR would be patch over rather than a point fix.

Re: Three people with inherited diseases successfully treated with CRISPR

#14

It's interesting that they did not just undo the inherited disease. I assumed that, especially with Sickle Cell where we have a good understanding of how it works, they would go into Chromosome 11 and put it back how it "should" be with CRISPR. But instead they apply a workaround, ensuring continued fetal haemoglobin production. The article does not mention whether that's because putting Chromosome 11 back with CRISP…

That's also what struck me as odd, but having maintained legacy systems for a long time I can see why it's sometimes preferable to use a proven if inelegant workaround than going in and try to fix the actual defect, especially if you have limited debugging options.

Analogies to IT and development aren’t that appropriate here tbh.

This isn’t a legacy system, if you want to keep the IT analogy this would be failing over to a different system or replacing an existing solution that doesn’t work with a competitive alternative rather than fixing all your bugs.

Re: Three people with inherited diseases successfully treated with CRISPR

#15
post #6

Earlier quoted context omitted.

You don't even need to switch fields. There are plenty of biomedical companies (and what isn't a tech company these days?) and projects that could use some passionate technical people.

What are the biomedical FAANGs?

Regeneron, Genetech, Amgen, Vertex, Gilead, Biogen

Re: Three people with inherited diseases successfully treated with CRISPR

#16

Earlier quoted context omitted.

That's also what struck me as odd, but having maintained legacy systems for a long time I can see why it's sometimes preferable to use a proven if inelegant workaround than going in and try to fix the actual defect, especially if you have limited debugging options.

Analogies to IT and development aren’t that appropriate here tbh. This isn’t a legacy system, if you want to keep the IT analogy this would be failing over to a different system or replacing an existing solution that doesn’t work with a competitive alternative rather than fixing all your bugs.

This wasn't meant very serious, i'm sorry I couldn't indicate that better. I'm in no way,shape or form qualified to chime in on this topic.

Re: Three people with inherited diseases successfully treated with CRISPR

#18

This is such mind-blowing success, it makes me contemplate switching fields. Is it reasonably feasible to switch from IT to Biotech/Medical Research in the mid thirties? I had a friend with Fukutin-related limb-girdle muscular dystrophy R13 and am wondering if CRISPR could be the solution for her.

It’s worth knowing there are very few diseases that can be cured with singular gene edits. Many diseases involve multiple genes, cascades of gene expression, or complex physiological pathways. Oh, and not to mention epigenetics and environmental factors.

Re: Three people with inherited diseases successfully treated with CRISPR

#19

I wonder since fetal hemoglobin has stronger binding with oxygen which means the blood can oxygenate faster if this can be used for other issues as well like reduced lung capacity and performance enhancement.

Yes but it also holds onto oxygen more tightly, making it difficult to released to cells that need it. There's no performance enhancement here.

Re: Three people with inherited diseases successfully treated with CRISPR

#20

This is such mind-blowing success, it makes me contemplate switching fields. Is it reasonably feasible to switch from IT to Biotech/Medical Research in the mid thirties? I had a friend with Fukutin-related limb-girdle muscular dystrophy R13 and am wondering if CRISPR could be the solution for her.

Anecdotal, but my cousin did exactly this. I will note, he is probably the most exceptional person I known of, beyond those classic geniuses everyone knows.
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