New podcast 🚨
🎙️ Movement disorders and mRNA therapy in Arginosuccinic aciduria 🎧

With Sonam Gurung and Julien Baruteau. UCL Great Ormond Street Institute of Child Health

Apple: podcasts.apple.com/gb/podcast/jim…

SoundCloud: soundcloud.com/user-109006120…

Spotify: open.spotify.com/episode/3TloPm…

#mRNA

mRNA therapy corrects defective glutathione metabolism and restores ureagenesis in preclinical argininosuccinic aciduria
Gurung et al,
doi.org/10.1126/scitra…

Fantastic work thanks to an amazing and dedicated team, especially Sonam Gurung, Oskar Vilhelmsson Timmermand and Dany Perocheau. A great example of successful collaboration between academia and industry with Tim Witney & Moderna!

New pathophysiological insight, new non-invasive tool to assess the disease with positron emission tomography and new curative therapy with repeated systematic mRNA injections in a preclinical model of inherited metabolic disease, argininosuccinic aciduria.

Messenger RNA can successfully treat rare genetic liver diseases! Very proud of our publication in Science Translational Medicine, a collaboration between Tim Witney's lab King's College London, Moderna and my group UCL and Great Ormond Street Hospital for Children.
science.org/doi/10.1126/sc…

Congratulations @jbaruteau37171 and all ⁦⁦Ashley Boyle⁩ ⁦Joanne Ng 伍仲昕⁩ ⁦Raj Karda⁩ ⁦Alan Parker⁩ ⁦Dr. Nicole Paulk⁩ ⁦Ahad Rahim⁩ ⁦Ajay Mistry⁩ ⁦Joanna Davidge⁩

mRNA therapy! science.org/doi/10.1126/sc…

📢New publication from us, JulienBaruteau’s group Great Ormond Street Hospital for Children UCL and Moderna:

'mRNA therapy corrects defective glutathione
metabolism and restores ureagenesis in preclinical
argininosuccinic aciduria' published today in Science Translational Medicine

science.org/doi/10.1126/sc…

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