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...

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Detalles Bibliográficos
Autores principales: Chen, Chun-Yu, Tran, Dominic M., Cavedon, Alex, Cai, Xiaohe, Rajendran, Raj, Lyle, Meghan J., Martini, Paolo G.V., Miao, Carol H.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2020
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7178004/
https://www.ncbi.nlm.nih.gov/pubmed/32330871
http://dx.doi.org/10.1016/j.omtn.2020.03.015

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7178004/
https://www.ncbi.nlm.nih.gov/pubmed/32330871
http://dx.doi.org/10.1016/j.omtn.2020.03.015

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