Gene Editing Therapy Seemingly Cures the Incurable, Mars' Missing Water is Located & Revisiting the Stanford Marshmallow Test

Cool Stuff Ride Home

Reggie Risseeuw and Marques Pfaff

News, Tech News, Science, Society & Culture

4.6 • 732 Ratings

🗓️ 19 May 2025

⏱️ 19 minutes

🔗️ Recording | iTunes | RSS

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Summary

Infant with rare, incurable disease is first to successfully receive personalized gene therapy treatment We may finally know what happened to the missing water on Mars Revisiting a famous marshmallow experiment: Children more likely to delay gratification if peer promises to wait as well New Twist on Famous Marshmallow Test: What Happens When You Add a Second Kid? Contact the Show: [email protected] Learn more about your ad choices. Visit megaphone.fm/adchoices

Transcript

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0:00.0	Welcome to Cool Stuff Ride Home, where we bring you some of the more cool stories from around the globe.
0:07.2	He's Reggie Rizu. I'm Marcus Papp. Just a couple of bozos bringing you the news, at least if you believe our Apple reviews.
0:13.4	On today's episode, gene editing therapy appears to have cured the incurable.
0:18.6	We may have located the missing water on Mars. What does that mean in the
0:21.9	search for life and revisiting the Stanford Marshmallow test? All coming up on cool stuff.
0:28.2	Turning to Science Daily and research out of the National Institute of Health. A research team
0:33.3	supported by the NIH has developed and safely delivered a personalized gene editing therapy
0:39.7	to treat an infant with a life-threatening incurable genetic disease. The infant was diagnosed
0:46.0	with a rare condition carbamil phosphate synthetase 1 deficiency shortly after birth, also known as
0:52.1	CPS1, and has responded positively, reportedly, to the treatment.
0:56.5	The process from diagnosis to treatment took only six months and marks the first time the technology
1:02.5	has been successfully deployed to treat a human patient.
1:06.3	The technology used in this study was developed using a platform that could be tweaked to treat a wide range of genetic disorders
1:12.9	and opens the possibility of creating personalized treatments in other parts of the body.
1:18.2	A team of researchers at the Children's Hospital of Philadelphia and the Perilman School of Medicine at the University of Pennsylvania
1:24.8	developed the customized therapy using the gene editing platform
1:28.5	CRISPR. You've heard us reference that in the past on this very show. They corrected a specific
1:33.4	gene mutation in the baby's liver cells that led to the disorder. CRISPR is an advanced
1:38.5	gene editing technology that enables precise changes to DNA inside living cells. This is the first known case of a personalized
1:47.0	CRISPR-based medicine administered to a single patient and was carefully designed to target non-reproductive
1:53.5	cells so changes would only affect the patient. For Dr. Joni L. Rudder, director of the NIH's
1:59.9	National Center for Advancing Translational Sciences,
	...

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