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676 : Ex-Vivo Autologous Stem Cell Gene Therapy Clinical Trial for Mucopolysaccharidosis Type IIIA: Trial in Progress - NCT04201405

Researchers

Presenter

  • Jane L Kinsella

Principal Investigators

  • Robert F Wynn

  • Brian Bigger

  • Adrian J. Thrasher

  • Claire Booth

  • Karen Buckland

  • Natalia Izotova

  • Stewart Rust

  • Daniel Weisberg

  • Heather J Church

  • Kathryn L Brammeier

  • Karen L Tylee

  • Ceri Jones

  • Helena Lee

  • Laura Ford

  • Atusa Sadegholnejat

  • Stuart Ellison

  • Simon A Jones

Medical Centers

  • Paediatric Bone Marrow Transplant, Royal Manchester Children's Hospital, Manchester University NHS Foundation Trust, Manchester, United Kingdom

  • Royal Manchester Children's Hospital, Manchester, United Kingdom

  • Stem Cell and Neurotherapies, University of Manchester, Manchester, United Kingdom

  • Great Ormond Street Institute of Child Health, UCL, London, United Kingdom

  • University College London/Great Ormond Street Hospital, London, United Kingdom

  • Willink Biochemical Genetics Unit, Manchester Foundation Trust, Manchester, United Kingdom

  • Willink Biochemical Genetics Unit, Manchester Foundation Trust, Manchester, United Kingdom

  • Transplantation Laboratory, Manchester NHS Foundation Trust, Manchester, United Kingdom

  • Stem Cell and Leukaemia Proteomics Laboratory, The University of Manchester, Manchester, United Kingdom

Locations

  • United Kingdom

Companies

  • N/A

Study Components

Therapeutic Area

  • Genetic Disorder

Disease

  • Mucopolysaccharidosis 3A

Biomarkers

  • Cluster of Differentiation 34

  • Cerebrospinal fluid

Drug/Treatment

  • Busulfan

Outcome

  • N/A

Study Design

  • Open Label

Phase

  • I/II

Study Id's

  • NCT04201405

Sponsors

  • N/A

Result

  • N/A

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