STOP p rogression
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Transcript of STOP p rogression
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STOPprogression
PROTECT & REGROWremaining tissues
REPLACE damaged tissues
A Cure For WolframThree Steps 2.0
Fumi Urano, MD, PhD
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1. Stop the progression
FDA-approved drugs
Novel drugs
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Common Molecular Pathway
ER Calcium leakage
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Identify FDA-approved DRUGS that can prevent ER calcium
depletion-mediated Cell Death
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Urano F. Diabetes (2014)
Induced Pluripotent Stem Cells from Wolfram patients
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Drug A prevents apoptosis in Wolfram iPSC-derived neuronal cells
Lu et al. unpublished
TREATMENT
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High in Patients
Low in Non-Patients
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Drug A in a mouse model of Wolfram
hippocampus cerebellum midbrain brain stem cortex0
0.5
1
1.5
2
2.5
3
con watercon danko waterko dan
1
23
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2. Protect and Regrow remaining tissues
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What is MANF? Purified from Brain
Promotes Cell Growth
Prevents Cell Death
Retinitis Pigmentosa
Parkison’s disease
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MANF-based treatment
Eye cells derived from Wolfram iPS cells
MANF peptideFrom Amarantus
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3. Replace the damaged tissues
REGENERATIVE MEDICINE
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Brain Cells and Eye cells from skin cells
Skin fibroblasts
BRAIN
EYE
iPS Cells
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Replace disease-causing DNA with the healthy one
L 1 2 3 4 5 6 7 8
Expected size 877bp
Expected size 1268bp
5’ 3’
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STOPprogression
PROTECT & REGROWremaining tissues
REPLACE damaged tissues
A Cure For WolframThree Steps 2.0