Paolo G.V. Martini

Systemic Messenger RNA Therapy as a Treatment for Methylmalonic Acidemia

An Ding, Jessica L. Schneller, Andrea Frassetto et al. · Cell Reports · 2017 · 260 citations · Full text

Pharmacy, Molecular Biology, Pharmacology +16

Systemic messenger RNA as an etiological treatment for acute intermittent porphyria

Lei Jiang, Pedro Berraondo, Daniel Jericó et al. · Nature Medicine · 2018 · 180 citations · Full text

Life Sciences, Molecular Biology, Molecular Medicine +8

Systemic mRNA Therapy for the Treatment of Fabry Disease: Preclinical Studies in Wild-Type Mice, Fabry Mouse Model, and Wild-Type Non-human Primates

Xuling Zhu, Ling Yin, Matt Theisen et al. · The American Journal of Human Genetics · 2019 · 160 citations · Full text

Life Sciences, Molecular Biology, Molecular Medicine +17

Lipid nanoparticle-targeted mRNA therapy as a treatment for the inherited metabolic liver disorder arginase deficiency

Brian Truong, Gabriella Allegri, Xiaobo Liu et al. · Proceedings of the National Academy of Sciences · 2019 · 154 citations · Full text

Life Sciences, Biochemistry, Molecular Biology +17

Dual mRNA therapy restores metabolic function in long-term studies in mice with propionic acidemia

Lei Jiang, Ji-Sun Park, Ling Yin et al. · Nature Communications · 2020 · 125 citations · Full text

Life Sciences, Molecular Biology, Pharmacology +17