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: Efficacy Evaluation of Liver-Directed Gene Therapy in Fabry Mice

Researchers

Presenter

  • Azadeh Kia

Principal Investigators

  • Jenny McIntosh

  • Cecilia Rosales

  • Paniz Hosseini

  • Rose Sheridan

  • Justyna Spiewak

  • Kevin Mills

  • Romuald Corbau

  • Amit C Nathwani

Medical Centers

  • Freeline, Stevenage, England, United Kingdom

  • Department of Haematology, Cancer Institute, University College London, London, United Kingdom

  • Department of Haematology, UCL Cancer Institute, London, United Kingdom

  • Centre for Inborn Errors of Metabolism, UCL Translational Mass Spectrometry, London, United Kingdom

  • Katharine Dormandy Haemophilia Centre and Thrombosis Unit, Royal Free NHS Trust, London, United Kingdom

Locations

  • United Kingdom

Companies

  • N/A

Study Components

Therapeutic Area

  • Genetic Disorder

Disease

  • Fabry disease

Biomarkers

  • N/A

Drug/Treatment

  • N/A

Outcome

  • N/A

Study Design

  • N/A

Phase

  • NA

Study Id's

  • N/A

Sponsors

  • N/A

Result

  • N/A

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