Gene therapy saves baby from fatal condition

The Naked Scientists Podcast

Dr Chris Smith

Natural Sciences, Science, Science Radio, Naked Scientists, Health & Fitness, Engineering, Medicine, Technology, Life Sciences

4.6 • 958 Ratings

🗓️ 17 February 2023

⏱️ 32 minutes

🔗️ Recording | iTunes | RSS

🧾️ Download transcript

Summary

In the news this week, a UK toddler with a rare genetic disease is saved by gene therapy: we explain how it works. Cockatoos wow scientists by taking a toolkit along to solve a problem. And a 55 million year old penguin fossil... And it's a whopper that weighed in at 150 kilos! Like this podcast? Please help us by supporting the Naked Scientists

Transcript

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0:00.0	All engine running.
0:04.0	running.
0:05.0	I'm so genius.
0:06.0	Welcome.
0:07.0	It's the show where we bring science.
0:09.0	What that essentially means is
0:10.0	Discoveries,
0:11.0	advances, research, technology, unbelievable.
0:15.0	Without further ado, this is the naked scientist.
0:18.0	Hello and welcome to the naked scientists,
0:20.0	the show where we bring you the latest breakthroughs in science technology and
0:24.0	medicine with me James Titco and I'm Chris Smith coming up this week a UK toddler with a
0:28.7	rare genetic disease is saved by gene therapy we explain how works, cockatoo's wow scientists by taking a toolkit
0:36.0	along to solve a problem and a 55 million year old penguin fossil. And it's a whopper that weighed in at
0:42.2	150 kilos.
0:44.0	The Naked Scientists Podcast is powered by UKfast.co.uk. This week scientists announced that a toddler with a rare fatal genetic condition has been treated successfully with a new gene therapy
1:05.8	approach.
1:06.8	19 year old Teddy has MLD, that's metachromatic leukadistrophy.
1:11.3	It's an inherited disease linked to a defective gene that normally codes for an essential
1:15.7	enzyme that's called Aral sulfates A. Without this working, material builds up inside
1:20.8	certain cell types and it causes them to die. The developing
1:24.0	brain is particularly vulnerable and affected individuals become
	...

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