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Safety data support human testing of hematopoietic stem cell gene therapy for mucopolysaccharid I

(Mary Ann Liebert, Inc./Genetic Engineering News) 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.
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