Ex-Vivo Autologous Stem Cell Gene Therapy Clinical Trial For Mucopolysaccharidosis Type Iiia: Update On Phase I/Ii Clinical Trial
Jane Louise Kinsella,Simon Jones,Adrian J. Thrasher,Claire Booth,Karen F. Buckland,Natalia Izotova,Stewart Rust,Daniel Weisberg,Heather J. Church,Karen L. Tylee,Kathryn L. Brammeier,Ceri Jones,Helena Lee,Laura Ford,Atusa Sadegholnejat,Stuart Ellison,Brian W. Bigger,Robert F. Wynn MOLECULAR GENETICS AND METABOLISM（2021）
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