Jason Mast

And at six months, baby KJ is treated with the first dose. This is in February. It's not amazingly effective, the first one, because it's a very small dose. They want to be very careful. But then they go for a second dose a few weeks later. And then the third dose, not yet clear how the third dose has gone.

And at six months, baby KJ is treated with the first dose. This is in February. It's not amazingly effective, the first one, because it's a very small dose. They want to be very careful. But then they go for a second dose a few weeks later. And then the third dose, not yet clear how the third dose has gone.

But after the second dose, they were able to really lower the medications he had been on to control ammonia. And they were able to loosen his diet so he could start eating protein for the first time, really, and like growing. And they no longer expect to give him a liver transplant. And they're hoping he has a, you know, not cured, but a much more mild form of his condition.

But after the second dose, they were able to really lower the medications he had been on to control ammonia. And they were able to loosen his diet so he could start eating protein for the first time, really, and like growing. And they no longer expect to give him a liver transplant. And they're hoping he has a, you know, not cured, but a much more mild form of his condition.

But after the second dose, they were able to really lower the medications he had been on to control ammonia. And they were able to loosen his diet so he could start eating protein for the first time, really, and like growing. And they no longer expect to give him a liver transplant. And they're hoping he has a, you know, not cured, but a much more mild form of his condition.

There's basically a delivery vehicle that's required to get your gene editing machinery into the liver. And they use what's called a lipid nanoparticle, which is basically a tiny little soap bubble that's Very, very, very, very, very small. That's sort of the delivery vehicle. And then inside of that, you put basically two components.

There's basically a delivery vehicle that's required to get your gene editing machinery into the liver. And they use what's called a lipid nanoparticle, which is basically a tiny little soap bubble that's Very, very, very, very, very small. That's sort of the delivery vehicle. And then inside of that, you put basically two components.

There's basically a delivery vehicle that's required to get your gene editing machinery into the liver. And they use what's called a lipid nanoparticle, which is basically a tiny little soap bubble that's Very, very, very, very, very small. That's sort of the delivery vehicle. And then inside of that, you put basically two components.

There's a gene editing protein that's sort of a two-part protein. It nicks DNA, and it has a part that changes a single letter from one to the other. And then there's what's called a guide RNA, which is basically GPS coordinates that

There's a gene editing protein that's sort of a two-part protein. It nicks DNA, and it has a part that changes a single letter from one to the other. And then there's what's called a guide RNA, which is basically GPS coordinates that

There's a gene editing protein that's sort of a two-part protein. It nicks DNA, and it has a part that changes a single letter from one to the other. And then there's what's called a guide RNA, which is basically GPS coordinates that

So you have a GPS coordinate within the genome for which letter to switch, you have a protein that switches the letter, and then you have this sort of FedEx envelope of a soap bubble that gets it into the liver.

So you have a GPS coordinate within the genome for which letter to switch, you have a protein that switches the letter, and then you have this sort of FedEx envelope of a soap bubble that gets it into the liver.

So you have a GPS coordinate within the genome for which letter to switch, you have a protein that switches the letter, and then you have this sort of FedEx envelope of a soap bubble that gets it into the liver.

I feel like in general, in science journalism, we avoid words like miracle and like cure quite often. But it is like, it is amazing. This is not a thing that could have been done for most of, until like basically very, very, very recently. This is a thing that like... Researchers have been trying to do this being sort of, like, broadly treat these kinds of conditions for a very long time.

I feel like in general, in science journalism, we avoid words like miracle and like cure quite often. But it is like, it is amazing. This is not a thing that could have been done for most of, until like basically very, very, very recently. This is a thing that like... Researchers have been trying to do this being sort of, like, broadly treat these kinds of conditions for a very long time.

I feel like in general, in science journalism, we avoid words like miracle and like cure quite often. But it is like, it is amazing. This is not a thing that could have been done for most of, until like basically very, very, very recently. This is a thing that like... Researchers have been trying to do this being sort of, like, broadly treat these kinds of conditions for a very long time.

You can look back at, like, publications and how people talked in the 80s or 90s when they kind of thought they were on the threshold of being able to, you know, replace genes and do things like this, and they weren't, and it didn't work. And now you hear you have a baby like KJ, and we don't yet know what his life will hold.

You can look back at, like, publications and how people talked in the 80s or 90s when they kind of thought they were on the threshold of being able to, you know, replace genes and do things like this, and they weren't, and it didn't work. And now you hear you have a baby like KJ, and we don't yet know what his life will hold.

You can look back at, like, publications and how people talked in the 80s or 90s when they kind of thought they were on the threshold of being able to, you know, replace genes and do things like this, and they weren't, and it didn't work. And now you hear you have a baby like KJ, and we don't yet know what his life will hold.