Preclinical assessment of an optimized AAV-FVIII vector in mice and non-human primates for the treatment of hemophilia A

Extensive clinical data from liver-mediated gene therapy trials have shown that dose-dependent immune responses against the vector capsid may impair or even preclude transgene expression if not managed successfully with prompt immune suppression. The goal of this preclinical study was to generate an...

Descripción completa

Detalles Bibliográficos
Autores principales: Elkouby, Liron, Armour, Sean M., Toso, Raffaella, DiPietro, Marti, Davidson, Robert J., Nguyen, Giang N., Willet, Mallory, Kutza, Stephanie, Silverberg, Joseph, Frick, Jennifer, Crosariol, Marco, Wang, Yuhuan, Wang, Chuansong, High, Katherine A., Sabatino, Denise E., Anguela, Xavier M.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2021
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8666598/
https://www.ncbi.nlm.nih.gov/pubmed/34977269
http://dx.doi.org/10.1016/j.omtm.2021.11.005

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8666598/
https://www.ncbi.nlm.nih.gov/pubmed/34977269
http://dx.doi.org/10.1016/j.omtm.2021.11.005

Ejemplares similares