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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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slide1

A Cure For Wolfram

Three Steps 2.0

Fumi Urano, MD, PhD

REPLACE

damaged tissues

PROTECT & REGROW

remaining tissues

STOP

progression

slide2

FDA-approved drugs

1. Stop the progression

Novel drugs

common molecular pathway
Common Molecular Pathway

ER Calcium leakage

slide7

High in Patients

Low in Non-Patients

slide10

What is MANF?

  • Purified from Brain
  • Promotes Cell Growth
  • Prevents Cell Death
  • Retinitis Pigmentosa
  • Parkison’s disease
manf based treatment
MANF-based treatment

Eye cells derived

from Wolfram iPS cells

MANF peptide

From Amarantus

slide12

3. Replace the damaged tissues

REGENERATIVE MEDICINE

brain cells and eye cells from skin cells
Brain Cells and Eye cells from skin cells

Skin fibroblasts

EYE

BRAIN

iPS Cells

replace disease causing dna with the healthy one
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

slide16

A Cure For Wolfram

Three Steps 2.0

REPLACE

damaged tissues

PROTECT & REGROW

remaining tissues

STOP

progression