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DR Beth PAYNE

Fishing for answers to DBA. DR Beth PAYNE. Who am I?. Haematology Consultant at University College London Hospital/Research at UCL CI. Why am I here. Trained in Haematology in Glasgow and London 2006 went to Harvard Medical School in Boston, USA Became interested in studying DBA.

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DR Beth PAYNE

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  1. Fishing for answers to DBA DR Beth PAYNE

  2. Who am I? • Haematology Consultant at University College London Hospital/Research at UCL CI

  3. Why am I here • Trained in Haematology in Glasgow and London • 2006 went to Harvard Medical School in Boston, USA • Became interested in studying DBA

  4. Researching what goes wrong in DBA – WHAT DO WE KNOW? • Red blood cells die prematurely

  5. Researching what goes wrong in DBA – WHAT DO WE KNOW? • Mistakes or absence one copy of a group of genes • These genes code for ribosomal proteins US National library of Medicine

  6. Researching what goes wrong in DBA – WHAT WOULD WE LIKE TO KNOW? • Since ribosomes are needed in all cells, why does DBA only cause problems with red blood cells? • Can we use research to help us find new treatments ?

  7. How to answer our questions? • Patients and their families are the greatest teachers • Some experiments cannot be done on patients so we need to learn more using other laboratory models

  8. Zebrafish

  9. Why do we use zebrafish? RO Karlstrom and DA Kane A flipbook of zebrafish embryogenesis Development (1996) 123:461

  10. Why do we use zebrafish? 2mm

  11. Why do we use zebrafish?

  12. Zebrafish Embryo stained for Haemoglobin at 4 days

  13. Effects of Leucine in DBA zebrafish

  14. Growth of DBA zebrafish with leucine

  15. Use Red fluorescent DBA zebrafish Ongoing projects – using DBA zebrafish to find new drugs for DBA Assess candidate compounds in primary cells

  16. Acknowledgements DFCI/Harvard Medical School USA Tom Look Evisa Gjini UjwalPyati Hong Sun AratiKhanna-Gupta Nancy Berliner AnuNarla Ben Ebert UCL Maria Virgilio Greg Pietka YoungrockJoung Tariq Enver Steve Wilson David Linch St Marys/Imperial College Josu De La Fuente DBA Patients and families!!!

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