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Targeting Endoplasmic Reticulum
 for Combating Type 1 Diabetes



              Fumihiko Urano, MD, PhD
                   April 10, 2012
Three Step Formula

                           Treat

                     Identify

              Understand
International
   Registry




  Understand
Type 1 diabetes
Root cause ?
Understand T1D
 at the Molecular Level




Endoplasmic Reticulum
       Disease
Endoplasmic
Reticulum ?
DNA becomes PROTEIN in the ER
Healthy Endoplasmic Reticulum


            LIFE
Disruption of Healthy ER
 causes beta cell death
How ?
Identify DRUGS that can
 maintain HEALTHY ER
Induced Pluripotent Stem Cells
                 (iPS cells)


                                   Beta Cells



Skin fibroblasts     iPS Cells

                                    Neurons
Three Step Formula

                           Treat

                     Identify

              Understand
URANO LAB
     Karen Sargent
   Kohsuke Kanekura
      Takashi Hara
   Christine Oslowski
        Simin Lu
Bryan O’Sullivan-Murphy
       Victor Liu
     Mai Kanekura
      Mariko Hara

       Julie Zhu

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Urano

Editor's Notes

  1. Proteins are needed for the body to function properly. They are the basis of body structures, such as collagen, and are used to synthesize enzymes, such as the hormone insulin, and antibodies in cells. The genomic DNA is a blueprint for these proteins. The Genetic information is stored in the DNA. In the nucleus, DNA code is copied into a messenger RNA molecule. In the cytoplasm, the mRNA code is translated into amino acids. Proteins are necklaces of amino acids. However, proteins are not functional after they are synthesized. To be functional, newly synthesized proteins have to fold into proper three-dimensional structures. This process is called “Protein Folding”.