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...
Main Authors: | Antje Banning, Anna Zakrzewicz, Xin Chen, Steven J. Gray, Ritva Tikkanen |
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Format: | Article |
Language: | English |
Published: |
MDPI AG
2021-05-01
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Series: | Cells |
Subjects: | |
Online Access: | https://www.mdpi.com/2073-4409/10/5/1259 |
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