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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

#51

Post by refurb »

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…

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. Host versus graft disease. The old immune system will attack the new immune system cells.

Re: Three people with inherited diseases successfully treated with CRISPR

#52

Post by BurningFrog »

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…

You can think of this as "turning the immune system off and on again", IT Crowd style.

I've heard it discussed as a possible way to treat autoimmune diseases like MS.

Re: Three people with inherited diseases successfully treated with CRISPR

#53

Post by BurningFrog »

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…

For acne, I support this policy.

For serious conditions like those in the article, forcing sterilization seems real cruel in an age with safe and plentiful abortion.

Re: Three people with inherited diseases successfully treated with CRISPR

#54

Post by BurningFrog »

Paperweight wrote: post #8

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

As long as someone is willing to pay the $1-2B for the formal testing, and wait a few years for the paperwork to go through, I assume it'll work like any other procedure.

Re: Three people with inherited diseases successfully treated with CRISPR

#55

Post by crimsonalucard1 »

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?

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.

Re: Three people with inherited diseases successfully treated with CRISPR

#57

Post by sszz »

loeg wrote: post #45
akkawwakka wrote: post #18

Earlier quoted context omitted.

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.

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)

Re: Three people with inherited diseases successfully treated with CRISPR

#58

Post by theelous3 »

crimsonalucard1 wrote: post #55
Roritharr wrote: post #32

Earlier quoted context omitted.

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

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 serious pause. Probably the most serious pause of your life, but still. I wouldn't shy away too quickly. When you know, you know.

Re: Three people with inherited diseases successfully treated with CRISPR

#59

Post by enchiridion »

refurb wrote: post #51
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…

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?

Re: Three people with inherited diseases successfully treated with CRISPR

#60

Post by alistairSH »

BurningFrog wrote: post #53
sandworm101 wrote: post #38

Earlier quoted context omitted.

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…

For acne, I support this policy. For serious conditions like those in the article, forcing sterilization seems real cruel in an age with safe and plentiful abortion.

safe and plentiful abortion

Not in the US.

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