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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

#41

Post by simcop2387 »

34679 wrote: post #37

>The remaining bone marrow cells are killed by chemotherapy, then replaced by the edited cells. Interesting that they need to kill the remaining bone marrow cells. I wonder what happens when they skip that step. Do the modified cells still reproduce, resulting in a partially effective treatment? Are they targeted by the immune system and eliminated? Are they just out-reproduced by the normal cells and fade into irrel…

More than likely I'd imagine it's the last option. They can only produce so many of the edited cells. The body is going to have orders of magnitude more of the original ones present before the treatment that I think they'd just overwhelm the new ones. If you remove the original ones the new ones have a place to thrive and eventually get to normal levels

Re: Three people with inherited diseases successfully treated with CRISPR

#42

Post by Mvandenbergh »

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?

The chemo used to kill the remaining bone marrow has serious fertility implications anyway so I'm not sure this is an issue.

Re: Three people with inherited diseases successfully treated with CRISPR

#43

Post by adrianN »

sandworm101 wrote: post #38
williamdclt wrote: post #36

Earlier quoted context omitted.

Add to that all women willing to give up the ability to have children for the treatment, which might be sizeable

Because of accidental pregnancies, there are some treatments denied to all fertile women regardless of choice. Ussually they are those that cause horrible birth defects (limb reductions etc) or make pregnancy deadly to the mother. Given the links between oxygen and injuries like FAS, this hemoglobin treatment may be one of that group. Example: Acutane was once used to treat acne in teenagers. Many/most doctors didnt…

Permanent sterilization is an option for women too.

Re: Three people with inherited diseases successfully treated with CRISPR

#44

Post by theelous3 »

Roritharr wrote: post #32
theelous3 wrote: post #28

Earlier quoted context omitted.

comp sci in prestigious european uni. Moved to us in early 2000s. Big nationwide datacenter stuff. Decided to become doctor. Did doctory stuff and floated around medical for a while before switching to psyc. Currently forensic psych for infamous US prison. Found that rather than patient hop in regular psyc practice, or drug up people in asylums, with trial cases spanning years you can really deep dive a person and do…

Did he support a family during his transition or was he still single? Was money a problem in general along the way?

Married no kids, partner also successful. Given his talents and character I don't believe money was an issue.

Re: Three people with inherited diseases successfully treated with CRISPR

#45

Post by loeg »

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.

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.

Re: Three people with inherited diseases successfully treated with CRISPR

#46

Post by DubiousPusher »

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.

By "holy grail" you mean it would produce a better result or just satisfy someone's OCD?

Re: Three people with inherited diseases successfully treated with CRISPR

#47

Post by koheripbal »

34679 wrote: post #37

>The remaining bone marrow cells are killed by chemotherapy, then replaced by the edited cells. Interesting that they need to kill the remaining bone marrow cells. I wonder what happens when they skip that step. Do the modified cells still reproduce, resulting in a partially effective treatment? Are they targeted by the immune system and eliminated? Are they just out-reproduced by the normal cells and fade into irrel…

That depends on the nature of the genetic disease.

If the original gene produces a protein that actively causes an issue in the body, versus if the gene produces a broken protein that simply fails to do its job.

In the first case, a partial replacement means that the original active protein continues to cause the disease.

In the second case, a partial replacement means that the repaired protein begins expressing itself.

In either case, there may or may not be a linear correlation between the percentage of fixed proteins and the severity of the disease, and that relationship probably isn't well understood in most disease cases, but a partial replacement is likely much more beneficial in the second case, rather than the first.

That is why these initial test are done on chemo patients, so that we can isolate testing of the method of treatment without needed to know what amount of the original tissue needs to be replaced.

There is still a long complicated road ahead. Not a lot of organs get fully replaced in other procedures.

Re: Three people with inherited diseases successfully treated with CRISPR

#48

Post by [deleted] »

theelous3 wrote: post #20
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.

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.

[deleted]

Re: Three people with inherited diseases successfully treated with CRISPR

#49

Post by Tuna-Fish »

DubiousPusher wrote: post #46
teekert wrote: post #26

Earlier quoted context omitted.

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.

By "holy grail" you mean it would produce a better result or just satisfy someone's OCD?

"Holy grail" not because of how it would work on this particular disease, but because it would allow basically limitless editing of any gene in any living human, allowing us to fix all hereditary genetic diseases, and do a whole lot of other things besides.

Re: Three people with inherited diseases successfully treated with CRISPR

#50

Post by belorn »

Roritharr wrote: post #4
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…

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.

It actually follow a common pattern in evolution. Doing big changes are hard, difficult and risky. Tinkering and doing small changes over time works for most things and any legacy system, as long they are not directly harmful, can remain as a byproduct. There is even a word for it in biology called spandrel.
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