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Three people with inherited diseases successfully treated with CRISPR

newscientist.com

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Re: Three people with inherited diseases successfully treated with CRISPR

#61

Post by patall »

akkawwakka wrote: post #18
Roritharr wrote: post #2

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.

Actually, there is a couple thousand monogenic Mendelian diseases, so work isn't short, also not people, just money. However, you won't treat a developmental delay with a gene edit, that only works for non-working enzymes etc. without too much permanent damage.

Re: Three people with inherited diseases successfully treated with CRISPR

#62

Post by patall »

teekert wrote: post #26
tialaramex wrote: post #3

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…

It is much easier to wreck things with Crisper (in this case the regulatory region that turns of fetal hemoglobin) that to really go in and alter one or a couple of specific base pairs. What they did is nice but the latter would be the holy grail.

If the holy grail is only a few years away, then yes. Normal CRISPR editing rely on non-homologous-end-joining (NHEJ) which is not an absolutely exact process. However, there are now base editors (look up prime editing) that edit a single position with very high specificity. Takes a few years from basic research to the clinics but they will be everywhere soon.

Re: Three people with inherited diseases successfully treated with CRISPR

#63

Post by refurb »

enchiridion wrote: post #59
refurb wrote: post #51

Earlier quoted context omitted.

If you don't kill off the existing bone marrow, you end up with a couple problems: 1. You only give the patient a small number of edited cells. You want those cells to multiply and replace the existing cells. If you don't remove the existing cells, the edited cells aren't going to reproduce very fast because the body has a feedback loop and is saying "we have enough bone marrow cells now, so no need to make more". 2.…

Do these patients have to be revaccinated?

I’m not a doctor, but I would say yes, you’d lose any acquired immunity since you kill off all the memory b-cells that store that information.

But you’d also gain the donor’s immunity, and if they’ve been vaccinated, the recipient would have that immunity as well.

Re: Three people with inherited diseases successfully treated with CRISPR

#64

Post by perl4ever »

DrAwdeOccarim wrote: post #15
person_of_color wrote: post #10

Earlier quoted context omitted.

What are the biomedical FAANGs?

Regeneron, Genetech, Amgen, Vertex, Gilead, Biogen

I've heard of those, but it's interesting to me that you list those and not, say, Illumina.

Re: Three people with inherited diseases successfully treated with CRISPR

#65

Post by rnet85 »

refurb wrote: post #63
enchiridion wrote: post #59

Earlier quoted context omitted.

Do these patients have to be revaccinated?

I’m not a doctor, but I would say yes, you’d lose any acquired immunity since you kill off all the memory b-cells that store that information. But you’d also gain the donor’s immunity, and if they’ve been vaccinated, the recipient would have that immunity as well.

That is not correct. Trained B cells are predominantly stored in lymph nodes. You also don’t gain host immunity by marrow transfer.

Re: Three people with inherited diseases successfully treated with CRISPR

#66

Post by milkcircle »

im3w1l wrote: post #31

Fetal hemoglobin binds stronger to oxygen than normal hemoglobin, so that a fetus can "steal" oxygen from its mother's blood. But if the mother has been crispred to also have fetal hemoglobin, then this won't work right? Meaning it's a male only treatment?

Hi. I do research pertaining to this disease and can answer your very thoughtful question. It is in fact safe for pregnant women to have high levels of fetal hemoglobin. There is a condition known as Hereditary Persistence of Fetal Hemoglobin (HPFH) in rare individuals who express near 100% fetal hemoglobin into adulthood, and they are able to undergo pregnancy perfectly normally with no harm to mother or child. While it is true that HbF has a mildly higher affinity to oxygen, it has not been shown in population-based studies that having higher levels of HbF by itself causes any actual clinical adverse events.

Re: Three people with inherited diseases successfully treated with CRISPR

#67

Post by loeg »

sszz wrote: post #57
loeg wrote: post #45

Earlier quoted context omitted.

My family has one of the well-known single-site cancer-causing mutations; we know what the specific site is, even. It would be immense to "fix" (I am not a geneticist) that single site. Even just for my kids.

Why would you prefer the risks of editing versus the risks of preimplantation screening? Editing would be strictly riskier unless there’s really no alternative (unlikely but possible)

Preimplantation screening isn't an option for me or any other of my family members who are long past implantation. I'm not saying I'd hop on stage 1 clinical trials of a potential editing cure, and we'd have to see what the data on risk looked like to compare it with the risk from cancer without editing.

Re: Three people with inherited diseases successfully treated with CRISPR

#68

Post by DrAwdeOccarim »

perl4ever wrote: post #64
DrAwdeOccarim wrote: post #15

Earlier quoted context omitted.

Regeneron, Genetech, Amgen, Vertex, Gilead, Biogen

I've heard of those, but it's interesting to me that you list those and not, say, Illumina.

I mean, they are a pick and shovel play. They don't make drugs? Plus, sequencing is great but it's getting saturated. Plus, ideas like direct sequencing using nanopores and chemical modification of nucleic acid to make the nanopore signal stronger may disrupt.

Re: Three people with inherited diseases successfully treated with CRISPR

#69

Post by DrAwdeOccarim »

person_of_color wrote: post #17
DrAwdeOccarim wrote: post #15

Earlier quoted context omitted.

Regeneron, Genetech, Amgen, Vertex, Gilead, Biogen

Where is 10x Genomics in the scheme of things?

They don't make drugs, so more of a pick and shovel play. More like ARM, Segate, WD, Micron?

Re: Three people with inherited diseases successfully treated with CRISPR

#70

Post by DrAwdeOccarim »

theelous3 wrote: post #58
crimsonalucard1 wrote: post #55

Earlier quoted context omitted.

I think you need to pause before considering this route. Are you that exceptional? This level of intelligence is incredibly rare and unless you're 100% sure you're capable of this, I wouldn't do it.

The guy has brains to burn of course, but tbh, it's simply about motivation. Medicine is a grind, more than an intellectual exercise. If you have the brains for actual tech there's no doubt you have the requisite intelligence. The only barrier is the stamina for the grind. I know I couldn't do it. Not as I am now. Maybe in the future. I couldn't say for sure, given I'm not clairvoyant. I agree that you should give se…

Very true. Not to discount MD training or doctors, but it's less about connecting disparate dots or learning how to solve problems and more about working out the brain like a muscle.
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