Treatment of Hemophilia A Using Factor VIII Messenger RNA Lipid Nanoparticles

• Main Authors: Chun-Yu Chen (Author), Dominic M. Tran (Author), Alex Cavedon (Author), Xiaohe Cai (Author), Raj Rajendran (Author), Meghan J. Lyle (Author), Paolo G.V. Martini (Author), Carol H. Miao (Author)
• Format: Book
• Published: Elsevier, 2020-06-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

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...