Jason Mast

So baby KJ was born last summer. He was the Muldoon's fourth child.

So baby KJ was born last summer. He was the Muldoon's fourth child.

So baby KJ was born last summer. He was the Muldoon's fourth child.

They're told that he's quite healthy and we're going to put him in the NICU for now, but, you know, he'll be back with you very soon. And then basically within 48 hours, a nurse pulls Kyle aside, the father, and pulls up KJ's arm and drops it down. And instead of flopping, as you would expect a baby's arm or anyone's arm, really, it's to do, it kind of shutters down.

They're told that he's quite healthy and we're going to put him in the NICU for now, but, you know, he'll be back with you very soon. And then basically within 48 hours, a nurse pulls Kyle aside, the father, and pulls up KJ's arm and drops it down. And instead of flopping, as you would expect a baby's arm or anyone's arm, really, it's to do, it kind of shutters down.

They're told that he's quite healthy and we're going to put him in the NICU for now, but, you know, he'll be back with you very soon. And then basically within 48 hours, a nurse pulls Kyle aside, the father, and pulls up KJ's arm and drops it down. And instead of flopping, as you would expect a baby's arm or anyone's arm, really, it's to do, it kind of shutters down.

And what they find is that his ammonia levels are in the thousands, when it should be, you know, like 10 or 20. And this is very dangerous.

And what they find is that his ammonia levels are in the thousands, when it should be, you know, like 10 or 20. And this is very dangerous.

And what they find is that his ammonia levels are in the thousands, when it should be, you know, like 10 or 20. And this is very dangerous.

So they rushed KJ across the street, basically, to the Children's Hospital of Philadelphia from UPenn Hospital. And they immediately put him on medicines to bring down that ammonia, put him on a strict diet. And they sequenced his genome. And they say, okay, what exactly is the issue here? And they find that he has a mutation in this one gene.

So they rushed KJ across the street, basically, to the Children's Hospital of Philadelphia from UPenn Hospital. And they immediately put him on medicines to bring down that ammonia, put him on a strict diet. And they sequenced his genome. And they say, okay, what exactly is the issue here? And they find that he has a mutation in this one gene.

So they rushed KJ across the street, basically, to the Children's Hospital of Philadelphia from UPenn Hospital. And they immediately put him on medicines to bring down that ammonia, put him on a strict diet. And they sequenced his genome. And they say, okay, what exactly is the issue here? And they find that he has a mutation in this one gene.

But what they realize is that this is actually a mutation that might be editable, that we might be able to make a gene editing treatment for.

But what they realize is that this is actually a mutation that might be editable, that we might be able to make a gene editing treatment for.

But what they realize is that this is actually a mutation that might be editable, that we might be able to make a gene editing treatment for.

For the last couple years, they had been basically preparing for a baby like KJ. Because there had been all these advancements in gene editing over the last decade. Many of your listeners probably have heard vaguely of CRISPR. There's one drug already approved for sickle cell. There's more in the works.

For the last couple years, they had been basically preparing for a baby like KJ. Because there had been all these advancements in gene editing over the last decade. Many of your listeners probably have heard vaguely of CRISPR. There's one drug already approved for sickle cell. There's more in the works.

For the last couple years, they had been basically preparing for a baby like KJ. Because there had been all these advancements in gene editing over the last decade. Many of your listeners probably have heard vaguely of CRISPR. There's one drug already approved for sickle cell. There's more in the works.

But the advancements had come to the point where you could make these really fine-tuned changes in DNA. And that... both creates some opportunities and some challenges. And the opportunity is there you can like treat as, you know, happened with KJ ultimately.

But the advancements had come to the point where you could make these really fine-tuned changes in DNA. And that... both creates some opportunities and some challenges. And the opportunity is there you can like treat as, you know, happened with KJ ultimately.