Jason Mast
👤 PersonAppearances Over Time
Podcast Appearances
We don't yet know exactly how well this works, but, like, the early signs are very promising, and that is... It is incredible, and it comes out of all of this gritty, biochemical, unsexy work and all this funding over all these years that have now produced this.
We don't yet know exactly how well this works, but, like, the early signs are very promising, and that is... It is incredible, and it comes out of all of this gritty, biochemical, unsexy work and all this funding over all these years that have now produced this.
We don't yet know exactly how well this works, but, like, the early signs are very promising, and that is... It is incredible, and it comes out of all of this gritty, biochemical, unsexy work and all this funding over all these years that have now produced this.
Are other people getting in line? I mean, so this is the big question. And it's sort of the more pessimistic, like the easy thing to do, sort of like if you're skeptical, is to look at this and be like, amazing, exciting. Love that this happened for KJ. Researchers pulled off something incredible. Not repeatable. You can't do it again. It's not going to work. And they have a really valid point.
Are other people getting in line? I mean, so this is the big question. And it's sort of the more pessimistic, like the easy thing to do, sort of like if you're skeptical, is to look at this and be like, amazing, exciting. Love that this happened for KJ. Researchers pulled off something incredible. Not repeatable. You can't do it again. It's not going to work. And they have a really valid point.
Are other people getting in line? I mean, so this is the big question. And it's sort of the more pessimistic, like the easy thing to do, sort of like if you're skeptical, is to look at this and be like, amazing, exciting. Love that this happened for KJ. Researchers pulled off something incredible. Not repeatable. You can't do it again. It's not going to work. And they have a really valid point.
The researchers involved here won't say how much it costs. It definitely costs in the millions of dollars. There's no one pulling up that money to make that at scale for thousands of infants or even hundreds of infants or even dozens of infants. This was supported in part by the NIH, who helped do some of the manufacturing for the monkey studies, if I recall correctly.
The researchers involved here won't say how much it costs. It definitely costs in the millions of dollars. There's no one pulling up that money to make that at scale for thousands of infants or even hundreds of infants or even dozens of infants. This was supported in part by the NIH, who helped do some of the manufacturing for the monkey studies, if I recall correctly.
The researchers involved here won't say how much it costs. It definitely costs in the millions of dollars. There's no one pulling up that money to make that at scale for thousands of infants or even hundreds of infants or even dozens of infants. This was supported in part by the NIH, who helped do some of the manufacturing for the monkey studies, if I recall correctly.
And the NIH is currently facing massive funding cuts. And so where is the money, where is the process going to come to do this at scale? Also, I should add, like, you can only kind of—you can only do this— Basically with a couple different types of conditions. You can do it with conditions that affect the liver.
And the NIH is currently facing massive funding cuts. And so where is the money, where is the process going to come to do this at scale? Also, I should add, like, you can only kind of—you can only do this— Basically with a couple different types of conditions. You can do it with conditions that affect the liver.
And the NIH is currently facing massive funding cuts. And so where is the money, where is the process going to come to do this at scale? Also, I should add, like, you can only kind of—you can only do this— Basically with a couple different types of conditions. You can do it with conditions that affect the liver.
You can do it, it's more complicated, but you can do it with conditions that affect the blood. Pretty much anything else, researchers are not yet at a place where they can reliably do this kind of gene editing work. And so for now, this won't be a one-off, but there won't be that many patients who benefit from it in the next few years, probably not. Yeah.
You can do it, it's more complicated, but you can do it with conditions that affect the blood. Pretty much anything else, researchers are not yet at a place where they can reliably do this kind of gene editing work. And so for now, this won't be a one-off, but there won't be that many patients who benefit from it in the next few years, probably not. Yeah.
You can do it, it's more complicated, but you can do it with conditions that affect the blood. Pretty much anything else, researchers are not yet at a place where they can reliably do this kind of gene editing work. And so for now, this won't be a one-off, but there won't be that many patients who benefit from it in the next few years, probably not. Yeah.
There's someone who's like, no, they should not have treated KJ. But there are, there will be, and there have to be discussions around what is the best use of our resources? Should we be doing these N of 1 cases versus trying to find ways of treating large swaths of KJ?
There's someone who's like, no, they should not have treated KJ. But there are, there will be, and there have to be discussions around what is the best use of our resources? Should we be doing these N of 1 cases versus trying to find ways of treating large swaths of KJ?
There's someone who's like, no, they should not have treated KJ. But there are, there will be, and there have to be discussions around what is the best use of our resources? Should we be doing these N of 1 cases versus trying to find ways of treating large swaths of KJ?
diseases, if it's going to be so resource intensive for one patient, there's going to have to be considerations about, you know, you can, the less testing you require, the more patients you can treat. So there's going to have to be discussions around how much testing you do and what is safe and what is a good bar for efficacy and who pays for this, which diseases are
diseases, if it's going to be so resource intensive for one patient, there's going to have to be considerations about, you know, you can, the less testing you require, the more patients you can treat. So there's going to have to be discussions around how much testing you do and what is safe and what is a good bar for efficacy and who pays for this, which diseases are