Gengineering
May. 15th, 2025 02:04 pm![[personal profile]](https://www.dreamwidth.org/img/silk/identity/user.png){kind=small}
ysabetwordsmithBaby Is Healed With World’s First Personalized Gene-Editing Treatment
Instead, KJ has made medical history. The baby, now 9 ½ months old, became the first patient of any age to have a custom gene-editing treatment, according to his doctors. He received an infusion made just for him and designed to fix his precise mutation.
THIS is what gengineering is for. It's also why research into human genetics and gengineering is essential.
More than 30 million people in the United States have one of more than 7,000 rare genetic diseases. Most are so rare that no company is willing to spend years developing a gene therapy that so few people would need.
This is why capitalism is bad for medicine in particular, science in general, and often people in general. A lot of really important things are not profitable, and if profit is the only motive, then those things don't get done and human quality of life suffers.
But KJ’s treatment — which built on decades of federally funded research — offers a new path for companies to develop personalized treatments without going through years of expensive development and testing.
This is why government funding of research is important. But don't rely on that as the only support because then they ignore all the topics and people they've decided to hate, which is why there's so little research into the needs of women, black people, etc.
While KJ’s treatment was customized so CRISPR found just his mutation, the same sort of method could be adapted and used over and over again to fix mutations in other places on a person’s DNA. Only the CRISPR instructions leading the editor to the spot on the DNA with the mutation would need to be changed. Treatments would be cheaper, “by an order of magnitude at least,” Dr. Marks said.
It takes time to set up a Nobel nomination, because you have to prove widespread impact, but I'd put this on the watchlist for nomination later.
It eventually could also be used for more common genetic disorders like sickle cell disease, cystic fibrosis, Huntington’s disease and muscular dystrophy.
Now there's a tricky ethical issue for you:
* The same gene that causes sickle cell anemia with two copies, also boosts malaria resistance with one copy.
* We have the technology to detect who has how many copies of which genes.
* We now have the technology to edit out the sickle-cell/malaria resistance gene.
* The relationship is so tight that prevalence of the gene and of malaria map exactly, except where human migration has caused some scatter.
* Malaria is expanding its range as climate change permits its carrier mosquitoes to travel farther.
Given these factors, what are your ethical assessments of the situation?
Mine would be:
* If you're concerned about the risk of having a baby with two copies of the sickle-cell/malaria resistance gene, get genetic testing for yourself and your partner before free-range procreation. If you both have a copy, the statistics are 1:4 with 0 copies or with 2 copies, and 2:4 for one copy (which does not cause sickle-cell anemia and does boost malaria resistance). In that case, lab-assisted procreation would be advisable to avoid doubling. If you choose to have children with a single copy, make sure they know that superpower has a drawback of risking double copies in free-range reproduction, so partner testing is advisable.
* Removing the sickle-cell/malaria resistance gene from the human gene pool would be inadvisable, especially at a time when the need for a single copy is growing. We know this, because evolution already did the math to generate an answer to the ethical question: Is the single copy worth the risk of throwing a double? The answer is yes, IF you live where malaria is prevalent, and that is why the gene map and disease map match so tightly.
Instead, KJ has made medical history. The baby, now 9 ½ months old, became the first patient of any age to have a custom gene-editing treatment, according to his doctors. He received an infusion made just for him and designed to fix his precise mutation.
THIS is what gengineering is for. It's also why research into human genetics and gengineering is essential.
More than 30 million people in the United States have one of more than 7,000 rare genetic diseases. Most are so rare that no company is willing to spend years developing a gene therapy that so few people would need.
This is why capitalism is bad for medicine in particular, science in general, and often people in general. A lot of really important things are not profitable, and if profit is the only motive, then those things don't get done and human quality of life suffers.
But KJ’s treatment — which built on decades of federally funded research — offers a new path for companies to develop personalized treatments without going through years of expensive development and testing.
This is why government funding of research is important. But don't rely on that as the only support because then they ignore all the topics and people they've decided to hate, which is why there's so little research into the needs of women, black people, etc.
While KJ’s treatment was customized so CRISPR found just his mutation, the same sort of method could be adapted and used over and over again to fix mutations in other places on a person’s DNA. Only the CRISPR instructions leading the editor to the spot on the DNA with the mutation would need to be changed. Treatments would be cheaper, “by an order of magnitude at least,” Dr. Marks said.
It takes time to set up a Nobel nomination, because you have to prove widespread impact, but I'd put this on the watchlist for nomination later.
It eventually could also be used for more common genetic disorders like sickle cell disease, cystic fibrosis, Huntington’s disease and muscular dystrophy.
Now there's a tricky ethical issue for you:
* The same gene that causes sickle cell anemia with two copies, also boosts malaria resistance with one copy.
* We have the technology to detect who has how many copies of which genes.
* We now have the technology to edit out the sickle-cell/malaria resistance gene.
* The relationship is so tight that prevalence of the gene and of malaria map exactly, except where human migration has caused some scatter.
* Malaria is expanding its range as climate change permits its carrier mosquitoes to travel farther.
Given these factors, what are your ethical assessments of the situation?
Mine would be:
* If you're concerned about the risk of having a baby with two copies of the sickle-cell/malaria resistance gene, get genetic testing for yourself and your partner before free-range procreation. If you both have a copy, the statistics are 1:4 with 0 copies or with 2 copies, and 2:4 for one copy (which does not cause sickle-cell anemia and does boost malaria resistance). In that case, lab-assisted procreation would be advisable to avoid doubling. If you choose to have children with a single copy, make sure they know that superpower has a drawback of risking double copies in free-range reproduction, so partner testing is advisable.
* Removing the sickle-cell/malaria resistance gene from the human gene pool would be inadvisable, especially at a time when the need for a single copy is growing. We know this, because evolution already did the math to generate an answer to the ethical question: Is the single copy worth the risk of throwing a double? The answer is yes, IF you live where malaria is prevalent, and that is why the gene map and disease map match so tightly.
(no subject)
Date: 2025-05-15 07:32 pm (UTC)It annoys me no end that CRISPR is almost the same as my proposed solution to that.. but life happened, and I never went on to do my Ph.D which is when I was going to do it. With the intention of finding a way to reduce the cost of single case gene-engineered treatments.
But at least it got done, eventually! That's the main point. Which is going to turn what was a fiddly artisanal process to produce bespoke treatments, into something that can be turned out in hospital lab as a cut&paste solution with minimal 'tailoring' needed.
Yes ...
Date: 2025-05-15 07:45 pm (UTC)That's why any high-card assignment that can find multiple souls to take it will send 3-5 souls at a time on that mission. It's also why the really rare stuff takes so long to achieve, because if there's only one soul at a time who can make a try for it, and they get knocked off course, you have to wait for another qualified soul to try again.
It's also why I look the vast swaths of humanity who are too poor and disadvantaged to achieve anything and I see billions of assignments going unfulfilled for lack of opportunity. The soul who's supposed to find the cure for cancer could be picking over a garbage dump in Delhi, and that does nobody any good. >_< Most people look at the poor as valueless mouths to feed. But when you know the metaphysics of advancement, you just see a crying waste of human resources.