Biosafety studies of Hematopoietic Stem Cell Gene Therapy for Mucopolysaccharidosis I shows promise for human trials

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… Continue reading Biosafety studies of Hematopoietic Stem Cell Gene Therapy for Mucopolysaccharidosis I shows promise for human trials