Young mice administered adult doses of AAV5-hFVIII-SQ achieve therapeutic factor VIII expression into adulthood

Valoctocogene roxaparvovec (AAV5-hFVIII-SQ) gene transfer provided reduced bleeding for adult clinical trial participants with severe hemophilia A. However, pediatric outcomes are unknown. Using a mouse model of hemophilia A, we investigated the effect of vector dose and age at treatment on transgen...

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Detalles Bibliográficos
Autores principales: Zhang, Lening, Yates, Bridget, Murphy, Ryan, Liu, Su, Xie, Lin, Handyside, Britta, Sihn, Choong-Ryoul, Bouwman, Taren, Galicia, Nicole, Tan, Danielle, Fonck, Carlos, Arens, Jeremy, Clark, Annie, Zhang, Weiming, Chandra, Sundeep, Srimani, Jaydeep, Holcomb, Jennifer, Van Tuyl, Andrea, Henshaw, Joshua, Vettermann, Christian, Siso, Silvia, Su, Cheng, Bullens, Sherry, Bunting, Stuart, O’Neill, Charles, Fong, Sylvia
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2022
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9440360/
https://www.ncbi.nlm.nih.gov/pubmed/36092364
http://dx.doi.org/10.1016/j.omtm.2022.08.002

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9440360/
https://www.ncbi.nlm.nih.gov/pubmed/36092364
http://dx.doi.org/10.1016/j.omtm.2022.08.002

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