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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

Gene TherapiesXenotransplantationEngineeringGeneticsGenetic EngineeringGene VectorSustained Transgene ExpressionSystems BiologyMedicineGene ExpressionGenome EditingMutated Pre SequenceMutagenesisGene Transfer