Publications by authors named "Alex Cavedon"

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The incidence of movement disorder increases with age and contrasts with subtle and limited neuroimaging abnormalities in argininosuccinic aciduria.
Sonam Gurung, Saketh Karamched, Dany Perocheau, Kiran K Seunarine, Tom Baldwin, Alex Cavedon

J Inherit Metab Dis· November 2024

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precision-cut liver slices model disease phenotype and monitor therapeutic response for liver monogenic diseases.
Dany Perocheau, Sonam Gurung, Loukia Touramanidou, Claire Duff, Garima Sharma, Alex Cavedon

F1000Res· April 2024

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mRNA therapy corrects defective glutathione metabolism and restores ureagenesis in preclinical argininosuccinic aciduria.
Sonam Gurung, Oskar Vilhelmsson Timmermand, Dany Perocheau, Ana Luisa Gil-Martinez, Magdalena Minnion, Alex Cavedon

Sci Transl Med· January 2024

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Induction of long-term tolerance to a specific antigen using anti-CD3 lipid nanoparticles following gene therapy.
Chun-Yu Chen, Amber Vander Kooi, Alex Cavedon, Xiaohe Cai, Jonathan Hoggatt

Mol Ther Nucleic Acids· December 2023

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Treatment of Hemophilia A Using Factor VIII Messenger RNA Lipid Nanoparticles.
Chun-Yu Chen, Dominic M Tran, Alex Cavedon, Xiaohe Cai, Raj Rajendran

Mol Ther Nucleic Acids· June 2020

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Systemic modified messenger RNA for replacement therapy in alpha 1-antitrypsin deficiency.
Ahmad Karadagi, Alex G Cavedon, Helen Zemack, Greg Nowak, Marianne E Eybye

Sci Rep· April 2020

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