Podcast: Gene therapy could be best hope to cure Duchenne muscular dystrophy

, | | August 3, 2020
unnamed file
Conner Curran, 9, and his brother Will, 7, playing on a swingset. Credit: Kholood Eid/NPR
This article or excerpt is included in the GLP’s daily curated selection of ideologically diverse news, opinion and analysis of biotechnology innovation.

Conner Curran was 4 when he was diagnosed with Duchenne muscular dystrophy. His muscles were already beginning to waste away. A doctor offered Conner’s parents some advice.

JESSICA CURRAN: Take your son home. Love him. Take him on trips while he’s walking. Give him a good life and enjoy him because there’s really not many options right now.

SHAPIRO: Today, Conner is 9 and not just walking, but running. The reason – an experimental gene therapy.

HAMILTON: Gene therapy seemed like the answer. After all, children with Duchenne muscular dystrophy lack a functional version of a gene called dystrophin, which helps muscles stay healthy.

JUDE SAMULSKI: The concept is very simple. You’re missing a gene, so you’re putting it back.

Follow the latest news and policy debates on agricultural biotech and biomedicine? Subscribe to our newsletter.
Related article:  Chronic brain injury CTE is only confirmed after death. How do we diagnose the living?

CONNER: I can run faster. I stand better, and I can walk to Goldberg’s. That’s a bagel shop. It’s more than two miles. And I couldn’t do that before.

HAMILTON: Conner’s body will never replace the muscle cells he lost, and it’s not clear how long his new genes will last. But the treatment seems to work. It’s now been tested on nine boys, and Pfizer is planning a much larger study for later this year. The therapy still has flaws, though. Several boys, including Conner, became ill temporarily after receiving the virus. Two ended up in the hospital. So Samulski’s been working on a fix.

Read the original post

Outbreak
Outbreak Daily Digest

podcasts GLP Podcasts More...
Biotech Facts & Fallacies
Talking Biotech
Genetics Unzipped

video Videos More...
stat hospitalai ink st x mod x

Meet STACI: STAT’s fascinating interactive guide to AI in healthcare

The Covid-19 pandemic underscores the importance of the technology in medicine: In the last few months, hospitals have used AI ...

bees and pollinators Bees & Pollinators More...
mag insects image superjumbo v

Disaster interrupted: Which farming system better preserves insect populations: Organic or conventional?

A three-year run of fragmentary Armageddon-like studies had primed the journalism pumps and settled the media framing about the future ...
dead bee desolate city

Are we facing an ‘Insect Apocalypse’ caused by ‘intensive, industrial’ farming and agricultural chemicals? The media say yes; Science says ‘no’

The media call it the “Insect Apocalypse”. In the past three years, the phrase has become an accepted truth of ...

infographics Infographics More...
breastfeeding bed x facebook x

Infographic: We know breastfeeding helps children. Now we know it helps mothers too

When a woman becomes pregnant, her risk of type 2 diabetes increases for the rest of her life, perhaps because ...

GMO FAQs GMO FAQs More...
biotechnology worker x

Can GMOs rescue threatened plants and crops?

Some scientists and ecologists argue that humans are in the midst of an "extinction crisis" — the sixth wave of ...
food globe x

Are GMOs necessary to feed the world?

Experts estimate that agricultural production needs to roughly double in the coming decades. How can that be achieved? ...
eating gmo corn on the cob x

Are GMOs safe?

In 2015, 15 scientists and activists issued a statement, "No Scientific consensus on GMO safety," in the journal Environmental Sciences ...
glp profiles GLP Profiles More...
Screen Shot at PM

Charles Benbrook: Agricultural economist and consultant for the organic industry and anti-biotechnology advocacy groups

Independent scientists rip Benbrook's co-authored commentary in New England Journal calling for reassessment of dangers of all GMO crops and herbicides ...
Screen Shot at PM

ETC Group: ‘Extreme’ biotechnology critic campaigns against synthetic biology and other forms of ‘extreme genetic engineering’

The ETC Group is an international environmental non-governmental organization (NGO) based in Canada whose stated purpose is to monitor "the impact of emerging technologies and ...
report this ad report this ad report this ad

Trending

News on human & agricultural genetics and biotechnology delivered to your inbox.
Optional. Mail on special occasions.
Send this to a friend