Cargando…

Knockout of the CMP–Sialic Acid Transporter SLC35A1 in Human Cell Lines Increases Transduction Efficiency of Adeno-Associated Virus 9: Implications for Gene Therapy Potency Assays

Recombinant adeno-associated viruses (AAV) have emerged as an important tool for gene therapy for human diseases. A prerequisite for clinical approval is an in vitro potency assay that can measure the transduction efficiency of each virus lot produced. The AAV serotypes are typical for gene therapy...

Descripción completa

Detalles Bibliográficos
Autores principales: Banning, Antje, Zakrzewicz, Anna, Chen, Xin, Gray, Steven J., Tikkanen, Ritva
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2021
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8160606/
https://www.ncbi.nlm.nih.gov/pubmed/34069698
http://dx.doi.org/10.3390/cells10051259