Extensive biosafety studies of hematopoietic stem cell (HSC) gene therapy, intended to replace a protein that patients with the inherited disease mucopolysaccaridosis I (MPS I) cannot produce, support clinical testing of the stem cell-based gene addition approach in MPS I patients. Evidence derived from these studies not only indicates that the HSC gene therapy is safe and well tolerated in mice, but also that it can produce sufficient amounts of the missing protein to affect MPS I without harming a patient's hematopoietic stem cells, according to an article in Human Gene Therapy.
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