Author
Moderna Therapeutics (United States)ORCID 0000-0002-3957-2984Unclaimed ORCID profile · follow to get notified of new preprints
4 papers
Recent·Most cited
Stephanie Yazinski, Susanna Canali, Yang Xu…·Patrick F. Finn
This paper demonstrates the use of LNP-delivered prime editors to correct the PAH R408W mutation in PKU models, achieving ~50% correction efficiency.
C. Chambers, Gillian L. Soo, Abbi L. Engel…·Timothy J. Cherry
This paper demonstrates reliable mRNA delivery via lipid nanoparticles to specific retinal cells in mice and human ocular tissues without significant inflammation.
Loukia Touramanidou, Sonam Gurung, Andrei Claudiu Cozmescu…·Julien Baruteau
This study shows that clodronate liposomes enhance lentiviral liver transduction but not AAV or LNP-mRNA in mice.
Sonam Gurung, Oskar Vilhelmsson Timmermand, Dany P.B. Perocheau…·Julien Baruteau
This study shows that mRNA therapy restores ureagenesis and glutathione metabolism in ASL-deficient mice, correcting liver disease and neurocognitive impairments.