Biochemical engraftment and clinical outcomes of ex-vivo autologous stem cell gene therapy for mucopolysaccharidosis type iiia
Anthea Guha,Robert Wynn,Brian Bigger,Jane Kinsella,Rebecca Holley,Jane Potter,Stuart Ellison,Adrian Thrasher, Kathryn Booth,Karen Buckland,Natalia Izotova,Stewart Rust,Daniel Weisberg,Heather Church,Karen Tylee,Kathryn Brammeier,Simon Jones,Helena Lee,Laura Ford,Atusa Sadegholnejat, Ceri Jones Blood（2023）
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gene therapy,stem cell,ex-vivo
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