4 • 1K Ratings
🗓️ 16 May 2025
⏱️ 7 minutes
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0:00.0 | Let's turn now to a promising medical breakthrough. |
0:03.0 | Doctors announced this week they have treated a newborn baby with a rare genetic disease using the world's first personalized gene editing treatment. |
0:12.0 | In the arms of his parents, Nicole and Kyle, KJ Muldoon looks much like any other happy, healthy nine-month-old baby. |
0:20.0 | But just days after he was born, his parents were initially given alarming news. |
0:25.6 | One of the doctors came to us and said, we think we know what's wrong. |
0:30.0 | Your son is very sick. |
0:31.7 | But the best place in the world for your child to be when he's very sick is next door. |
0:36.2 | Doctors at the Children's Hospital of Philadelphia told them that their son had a rare |
0:40.2 | genetic disease known as CPS1. |
0:43.4 | It only affects one in 1.3 million babies, but the disease caused by a single mutation in |
0:49.7 | his DNA meant their child's body couldn't process ammonia and doctors had to carefully restrict |
0:55.2 | protein in his diet. |
0:57.3 | You Google CPS1 deficiency and it's either fatality rate or liver transplant. |
1:03.0 | The choices were stark. |
1:04.1 | Our child is sick. |
1:06.3 | We either have to get a liver transplant or do this, given this medicine that's never been given anybody |
1:13.6 | before, right? I mean, what an impossible decision to make. |
1:17.6 | Doctors told them they could try a new experimental procedure with KJ, one that had been in the works for years. |
1:23.6 | Doctors used the gene editing technology known as CRISPR to isolate and correct |
1:29.0 | that single mutation. In essence, CRISPR technology was used as a kind of GPS to go into the |
1:34.9 | baby's DNA and use an enzyme to fix just one errant letter out of about $3 billion in his DNA. |
1:42.0 | Therapy as quickly as essential. Dr. Rebecca Arons Nicholas was one of the leaders of the team. |
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