An Empowered, Clinically-Viable Hematopoietic Stem Cell Gene Therapy Approach for the Treatment of Multisystemic Mucopolysaccharidosis Type II

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Scientists developed up to clinical readiness a new Hematopoietic Stem Cell (HSC) gene therapy approach for Mucopolysaccharidosis type II that benefitted from a novel highly effective transduction protocol.
[Molecular Therapy]
AbstractGraphical Abstract