Non-genotoxic conditioning facilitates hematopoietic stem cell gene therapy for hemophilia a using bioengineered factor viii

HIGHLIGHTS

• who: Athena L. Russell from the Emory University, Atlanta, GA, USA have published the research: Non-genotoxic conditioning facilitates hematopoietic stem cell gene therapy for hemophilia A using bioengineered factor VIII, in the Journal: (JOURNAL) of 21/06/2021
• what: The authors describe a protocol using an immunotoxin targeting CD117 (c-kit) to achieve endogenous hematopoietic stem cell depletion and a cocktail of monoclonal antibodies to provide transient immune suppression against the transgene product in a murine HA gene therapy model. This approach has potential advantages over in_vivo AAV delivery (e_g, vector integration allows stable . . .
  

   

  

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