Validation of a mutated PRE sequence allowing high and sustained transgene expression while abrogating WHV-X protein synthesis: Application to the gene therapy of WAS

M. A. Zanta-Boussif, S. Charrier, A. Brice-Ouzet, S. Martin, P. Opolon, A. J. Thrasher, T. J. Hope, A. Galy*

*Corresponding author for this work

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Biochemistry, Genetics and Molecular Biology