Alex G. Cavedon

Systemic modified messenger RNA for replacement therapy in alpha 1-antitrypsin deficiency

L. Rice, S. Strom, P. Martini, 2020, Scientific Reports.

Treatment of Hemophilia A Using Factor VIII Messenger RNA Lipid Nanoparticles

P. Martini, M. Lyle, C. Miao, 2020, Molecular therapy. Nucleic acids.

mRNA therapy restores ureagenesis and corrects glutathione metabolism in argininosuccinic aciduria

M. Ryten, L. Rice, P. Finn, 2022, bioRxiv.

Ex vivo primary liver sections recapitulate disease phenotype and therapeutic rescue for liver monogenic diseases

N. Sebire, L. Rice, P. Finn, 2023, bioRxiv.

Macrophage inhibitor clodronate enhances liver transduction of lentiviral but not AAV vectors or mRNA lipid nanoparticles in vivo

L. Rice, P. Finn, P. Martini, 2023, bioRxiv.

The incidence of movement disorders increases with age and contrasts with subtle and limited neuroimaging abnormalities in argininosuccinic aciduria

J. Clayden, T. Kalber, S. Santra, 2023, bioRxiv.