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Autologous CD34+ hematopoietic stem and progenitor cells expressing adenosine deaminase is an investigational lentiviral gene therapy. It comprises autologous CD34+ hematopoietic stem and progenitor cells (HSPCs) that had been genetically modified ex vivo with EFS-ADA LV.
Autologous CD34+ hematopoietic stem and progenitor cells expressing adenosine deaminase is an investigational lentiviral gene therapy. It comprises autologous CD34+ hematopoietic stem and progenitor cells (HSPCs) that had been genetically modified ex vivo with EFS-ADA LV.