Immunological aspects of immunodeficiency diseases part 59

Immune 3

 

GENE THERAPY FOR SCID

Long-term immune reconstitution has been achieved in patients with SCID caused by the common γ-chain deficiency or ADA deficiency, using gene therapy. This was achieved by ex vivo gene transfer to hematopoetic stem cells isolated from the patient’s bone marrow. These gene reconstituted stem cells were retransfused into the patient. To date, gene therapy has been restricted to patients without an HLA-matched family donor.

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