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ADA umbilical cord blood cells

Development stage: Unknown
Lead developer: University of California, Los Angeles
Modality: Cell Therapies, Gene Therapies
Administration: Intravenous

Overview

ADA umbilical cord blood cells are genetically modified hematopoietic stem cells derived from umbilical cord blood, engineered to express the normal human adenosine deaminase (ADA) gene. This therapy is used for the treatment of severe combined immunodeficiency (SCID) due to ADA deficiency. The treatment involves collecting CD34+ cells from the patient's umbilical cord blood, transducing them with a retroviral vector containing the normal human ADA gene, and then autologously transplanting these modified cells back into the patient. This approach allows for the expression of functional ADA enzyme in the patient's immune cells, potentially providing a therapeutic benefit for ADA-SCID patients.

Other names: ADA Umbilical Cord Blood Cells

Targets

ADA (Adenosine deaminase)

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