this post was submitted on 16 May 2025
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Infant becomes world's first patient to undergo personalized gene-editing treatment (www.cbsnews.com)
submitted 1 month ago* (last edited 1 month ago) by MicroWave@lemmy.world to c/health@lemmy.world
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Soon after KJ Muldoon was born in the summer of 2024, he was diagnosed with a rare genetic disorder that is fatal for about half the infants who are born with it.

Until now, the only effective long-term treatment for the rare metabolic disease known as severe Carbamoyl Phosphate Synthetase 1 deficiency, or CPS1, had been a liver transplant.

Instead, doctors at the Children's Hospital of Philadelphia told KJ's family they could try something never done before. They would use a technology known as CRISPR, a personalized gene-editing therapy, to find the one uniquely mutated gene out of 20,000 in his little body, and fix it.

He became the first known patient in the world to be treated using CRISPR personalized just for him, according to a news release from Penn Medicine. His case was published Thursday in the New England Journal of Medicine.

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[–] Scubus@sh.itjust.works 4 points 1 month ago (1 children)

I don't have an agenda, but the post states that it is the first case of personalized human gene editing. Was the chinese doctor's not "personalized"?

  1. Fair enough

  2. ... fair enough? It would seem that its either good or bad and the odds of death being high shouldn't effect whether it's a good or bad idea. If i misunderstanding lmk

  3. From my understanding, thats not really the issue. Humanity has decided that gene editing is not to be fucked with, have we not? I used to study gene editing and from everything I read and heard it would be universally illegal for me to edit an embryo and then implant it under any circumstances. Has that changed? Did I misunderstand in the first place?

I've always been very pro gene editing, but I want to to be approached as safely as possible. I'd prefer for it to only be done on consenting adults, and in such a way that any altered genes are non contagious and non hereditary. I guess I do kinda have a dog in this race, but only insofar as I don't want rogue scientists bringing negative press to a positive tool, or the resulting damage to life those rogue scientists can do.