Mucopolysachhridosis

Mucopolysachhridosis

Latest Posts

• 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…

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