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STOP p rogression

A Cure For Wolfram Three Steps 2.0 Fumi Urano, MD, PhD. REPLACE damaged tissues. PROTECT & REGROW remaining tissues. STOP p rogression. FDA-approved drugs. 1. Stop the progression. Novel drugs. Common Molecular Pathway. ER Calcium leakage.

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STOP p rogression

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  1. A Cure For Wolfram Three Steps 2.0 Fumi Urano, MD, PhD REPLACE damaged tissues PROTECT & REGROW remaining tissues STOP progression

  2. FDA-approved drugs 1. Stop the progression Novel drugs

  3. Common Molecular Pathway ER Calcium leakage

  4. Identify FDA-approved DRUGS that can prevent ER calcium depletion-mediated Cell Death

  5. Induced Pluripotent Stem Cells from Wolfram patients Urano F. Diabetes(2014)

  6. Drug A prevents apoptosis in Wolfram iPSC-derived neuronal cells TREATMENT Lu et al. unpublished

  7. High in Patients Low in Non-Patients

  8. Drug A in a mouse model of Wolfram 2 3 1

  9. 2. Protect and Regrow remaining tissues

  10. What is MANF? • Purified from Brain • Promotes Cell Growth • Prevents Cell Death • Retinitis Pigmentosa • Parkison’s disease

  11. MANF-based treatment Eye cells derived from Wolfram iPS cells MANF peptide From Amarantus

  12. 3. Replace the damaged tissues REGENERATIVE MEDICINE

  13. Brain Cells and Eye cells from skin cells Skin fibroblasts EYE BRAIN iPS Cells

  14. Replace disease-causing DNA with the healthy one 5’ 3’ L 1 2 3 4 5 6 7 8 Expected size 1268bp Expected size 877bp

  15. A Cure For Wolfram Three Steps 2.0 REPLACE damaged tissues PROTECT & REGROW remaining tissues STOP progression

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