Gene Therapy for Adenosine Deaminase Severe Combined Immune Deficiency Using Peripheral Blood and EFS ADA Vector

Conditions

• Adenosine Deaminase Severe Combined Immune Deficiency

Interventions

• COMBINATION_PRODUCT: A cryopreserved formulation of autologous mPB CD34+ hematopoietic stem and progenitor cells transduced ex vivo with the EFS-ADA lentiviral vector encoding the human ADA enzyme

Sponsor

University of California, Los Angeles