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: Flow Cytometry Assay to Monitor Effectiveness of Gene Therapy Correction in Peripheral Blood Mononuclear Cells of Gaucher Disease Type I Patients

Researchers

Presenter

  • Cecilia Barese

Principal Investigators

  • Michael Waring

  • Richard Pfeifer

  • Chris Mason

Medical Centers

  • AVROBIO Inc, Cambridge, MA

  • Ragon Institute of Massachusetts General Hospital, MIT, and Harvard, Cambridge, MA

  • Howard Hughes Medical Institute, Chevy Chase, MD

  • Advanced Centre for Biochemical Engineering, University College London, London, United Kingdom

Locations

  • United States

  • United Kingdom

Companies

  • N/A

Study Components

Therapeutic Area

  • Endocrine/metabolic Diseases (ME)

  • Genetic Disorder

Disease

  • Gaucher disease 1

Biomarkers

  • Peripheral blood mononuclear cell

  • Cluster of Differentiation 14

Drug/Treatment

  • beta-glucocerebrosidase

  • Fluorescein sodium

Outcome

  • N/A

Study Design

  • N/A

Phase

  • NA

Study Id's

  • N/A

Sponsors

  • N/A

Result

  • N/A

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