Nature Communications (Oct 2020)
Dual mRNA therapy restores metabolic function in long-term studies in mice with propionic acidemia
- Lei Jiang,
- Ji-Sun Park,
- Ling Yin,
- Rodrigo Laureano,
- Eric Jacquinet,
- Jinsong Yang,
- Shi Liang,
- Andrea Frassetto,
- Jenny Zhuo,
- Xinhua Yan,
- Xuling Zhu,
- Steven Fortucci,
- Kara Hoar,
- Cosmin Mihai,
- Christopher Tunkey,
- Vlad Presnyak,
- Kerry E. Benenato,
- Christine M. Lukacs,
- Paolo G. V. Martini,
- Lin T. Guey
Affiliations
- Lei Jiang
- Moderna Inc., 200 Technology Square
- Ji-Sun Park
- Moderna Inc., 200 Technology Square
- Ling Yin
- Moderna Inc., 200 Technology Square
- Rodrigo Laureano
- Moderna Inc., 200 Technology Square
- Eric Jacquinet
- Moderna Inc., 200 Technology Square
- Jinsong Yang
- Moderna Inc., 200 Technology Square
- Shi Liang
- Moderna Inc., 200 Technology Square
- Andrea Frassetto
- Moderna Inc., 200 Technology Square
- Jenny Zhuo
- Moderna Inc., 200 Technology Square
- Xinhua Yan
- Moderna Inc., 200 Technology Square
- Xuling Zhu
- Moderna Inc., 200 Technology Square
- Steven Fortucci
- Moderna Inc., 200 Technology Square
- Kara Hoar
- Moderna Inc., 200 Technology Square
- Cosmin Mihai
- Moderna Inc., 200 Technology Square
- Christopher Tunkey
- Moderna Inc., 200 Technology Square
- Vlad Presnyak
- Moderna Inc., 200 Technology Square
- Kerry E. Benenato
- Moderna Inc., 200 Technology Square
- Christine M. Lukacs
- Moderna Inc., 200 Technology Square
- Paolo G. V. Martini
- Moderna Inc., 200 Technology Square
- Lin T. Guey
- Moderna Inc., 200 Technology Square
- DOI
- https://doi.org/10.1038/s41467-020-19156-3
- Journal volume & issue
-
Vol. 11,
no. 1
pp. 1 – 10
Abstract
Propionic acidemia is a serious pediatric inherited disorder with no effective treatments. Here the authors demonstrate that delivering dual mRNAs as an enzyme replacement approach can be used as an effective therapy in a mouse model of propionic acidemia, with potential applicability to chronically administer multiple mRNAs in other genetic disorders.
