Treatment of Hemophilia A Using Factor VIII Messenger RNA Lipid Nanoparticles

Treatment of Hemophilia A Using Factor VIII Messenger RNA Lipid Nanoparticles

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 s...

Full description

Bibliographic Details
Main Authors: Chun-Yu Chen, Dominic M. Tran, Alex Cavedon, Xiaohe Cai, Raj Rajendran, Meghan J. Lyle, Paolo G.V. Martini, Carol H. Miao
Format: Article
Language:English
Published: Elsevier 2020-06-01
Series:Molecular Therapy: Nucleic Acids
Subjects:
factor VIII
hemophilia A
lipid nanoparticle
nonviral
gene delivery
gene transfer
Online Access:http://www.sciencedirect.com/science/article/pii/S2162253120301062

Internet

http://www.sciencedirect.com/science/article/pii/S2162253120301062

Similar Items