Ontology highlight
ABSTRACT:
SUBMITTER: Chen CY
PROVIDER: S-EPMC7178004 | biostudies-literature | 2020 Jun
REPOSITORIES: biostudies-literature
Chen Chun-Yu CY Tran Dominic M DM Cavedon Alex A Cai Xiaohe X Rajendran Raj R Lyle Meghan J MJ Martini Paolo G V PGV Miao Carol H CH
Molecular therapy. Nucleic acids 20200407
Hemophilia A (HemA) patients are currently treated with costly and inconvenient replacement therapy of short-lived factor VIII (FVIII) protein. Development of lipid nanoparticle (LNP)-encapsulated mRNA encoding FVIII can change this paradigm. LNP technology constitutes a biocompatible and scalable system to efficiently package and deliver mRNA to the target site. Mice intravenously infused with the luciferase mRNA LNPs showed luminescence signals predominantly in the liver 4 h after injection. R ...[more]