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Movement disorders and mRNA therapy in Arginosuccinic aciduria
Manage episode 398846665 series 3001064
Kandungan disediakan oleh JIMD Podcasts and Journal of Inherited Metabolic Disease. Semua kandungan podcast termasuk episod, grafik dan perihalan podcast dimuat naik dan disediakan terus oleh JIMD Podcasts and Journal of Inherited Metabolic Disease atau rakan kongsi platform podcast mereka. Jika anda percaya seseorang menggunakan karya berhak cipta anda tanpa kebenaran anda, anda boleh mengikuti proses yang digariskan di sini https://ms.player.fm/legal.
Dr Sonam Gurung and Dr Julien Baruteau discuss movement disorders in Arginosuccinic aciduria and explain how recent work with mRNA therapy shows potential as a treatment in this condition. The incidence of movement disorder increases with age and contrasts with subtle and limited neuroimaging abnormalities in argininosuccinic aciduria Gurung et al https://doi.org/10.1002/jimd.12691 mRNA therapy corrects defective glutathione metabolism and restores ureagenesis in preclinical argininosuccinic aciduria Gurung et al https://doi.org/10.1126/scitranslmed.adh1334
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