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Posted on 05/16/2025 1:29:18 PM PDT by nickcarraway
An American baby, KJ Muldoon, born with a rare and life-threatening genetic disease, is growing normally after undergoing the first successful personalized gene therapy.
In a new study, researchers report that the baby is among the first humans to receive this therapy to correct tiny yet critical errors in his genetic code. These errors are fatal to half of the affected infants. Although this treatment is not anticipated to become mainstream, doctors hope the technology will eventually aid millions with similar conditions.
Dr. Kiran Musunuru from the University of Pennsylvania said, “This is the first step towards the use of gene editing therapies to treat a wide variety of rare genetic disorders for which there are currently no definitive medical treatments.” Musunuru co-authored the study, examining the baby’s treatment.
The US baby is the first to undergo successful gene therapy. 350 million people suffer from similar conditions Experts claim that there are close to 350 million people around the world who suffer from rare diseases, much like the one the baby from Pennsylvania suffered from. Most of these diseases are genetic.
The case of the US baby is quite particular, as he was born with severe CPS1 deficiency. According to experts, this disease is estimated to affect one in a million babies. Infants who suffer from this condition usually lack an enzyme needed to help remove ammonia from their bodies. Others with the condition may require liver transplants. After learning of the odds KJ was facing, his parents, Kyle and Nicole, decided to undergo the experimental procedure due to their fear of losing their baby.
The treatment for KJ was designed in just six months
Before deciding to undertake the treatment, KJ’s parents weighed all of their options. Nicole explained to NBC News, “We were, like, you know, weighing all the options, asking all the questions for either the liver transplant, which is invasive, or something that’s never been done before.”
On the other hand, Kyle said, “We prayed, we talked to people, we gathered information, and we eventually decided that this was the way we were going to go.”
It took just six months for researchers at Children’s Hospital of Philadelphia and Penn Medicine, alongside their partners, to create the personalized gene therapy to treat KJ’s faulty gene. Researchers used CRISPR, a gene editing tool that won its inventors the Nobel Prize in 2020.
In February 2025, KJ received his first IV fusion with the gene editing therapy. It was delivered through fatty droplets called lipid nanoparticles, which are taken up by cells in the liver. After receiving two more fusions in March and April, KJ is now developing positively.
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Amazing.
I’m happy if the outcome is good for the baby but this stuff scares the heck out of me.
You’re not alone.
“...losing their baby...”
That has a specific meaning in English.
Was the treatment performed in utero?
But the LAST thing that I want to see happening is the creation of Khan Noonien Singh.
KHAAAAAAAAAN!
> Was the treatment performed in utero?
Nope, post birth.
I need no shortage of gene therapy for no shortage of old age conditions....
THIS is the appropriate use of mRNA technology, correcting existing defects, not creating them with mutagens mislabeled as “vaccines”.
It’s a rare condition yet 350 million people have it? Did I read this wrong?
Nope. Nope. Nope.
3 births a year in the US is considered rare.
“THIS is the appropriate use of mRNA technology”
THIS is not mRNA tech.
It does me, too, but I do know an older man who has an enzyme deficiency that has caused him problems his whole life and he volunteered to be a test subject for the procedure.
I guess they didn’t even know if he’d survive it not because it’s dangerous but it’s believer and not afraid of dying.
Most new treatments can be scary but five years from now it will be routine.
Out of 7 billion is rather rare.
It’s hard to tell from the original article if they are employing mRNA to deliver the CRISPR altered material.
But it does mention lipid nanoparticles, and those are used to encase mRNA so that your body’s enzymes don’t destroy it before it can do something useful. That was Robert Malone’s great discovery.
So it does sound like mRNA could be the delivery system that they are using. If it’s what I think they are doing, the mRNA would instruct the boy’s own defective liver cells to produce genetically corrected versions that can produce the missing enzyme.
The CRISPR part would precede this. It would start with one of his own liver cells, use CRISPR to add the missing enzyme sequence, and then use the resulting DNA to “express” the mRNA code for the corrected cell.
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