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Introduction to stem cells

Introduction to stem cells. Stem cell Community Forum. Raymond Wong, PhD Neuroregeneration Research Unit, CERA. What is a cell?. An organism’s basic unit of structure & function All living things are made of cells Cells contain information that is inherited (DNA/genes)

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Introduction to stem cells

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  1. Introduction to stem cells Stem cell Community Forum Raymond Wong, PhD Neuroregeneration Research Unit, CERA

  2. What is a cell? • An organism’s basic unit of structure & function • All living things are made of cells • Cells contain information that is inherited (DNA/genes) • Levels of organisation: • organism, organs, tissues, cells, DNA=> proteins

  3. What is a stem cell? From Stem Cell Teacher Kit, 2010 (Australian Stem Cell Centre) • Can self-renew: reproduce itself • Can differentiate: give rise to specialized cells Handbook of stem cells, Elsevier, R. Lanza, Editor

  4. Different types of stem cells: Multipotent vs pluripotent From Sarah Wray From Stem Cell Research Foundation

  5. Pluripotent stem cells ‘Reprogramming’ iPS cells adapted from P. Donovan Can proliferate indefinitely in the lab: unlimited cell source Pluripotent: can give rise to any cells type in the body

  6. Reprogramming to make iPS cells Human iPS cells Skin biopsy Adult fibroblasts Reprogramming Oct4 Hair Keratinocytes • ‘Switch on’ several genes => ‘add’ several proteins in the cells Nanog

  7. Potentials of iPS cells: Patient Cell replacement therapy Eye cells (or any cell type) Somatic cells Drug screening Disease modelling Patient-specific stem cells (iPS cells) Reprogramming Differentiation

  8. Case study: Leber’s hereditary optic neuropathy (LHON) Designed by Brian Ashton, 20 yrs old LHON patient (http://thegenetichouse.wordpress.com) • Inherited disease affecting ~ 1 in 30000 individuals, predominantly young males • Central vision loss occurs usually around teenage to early twenties • Mutations in mitochondria cause bioenergetics dysfunction • Characterized by loss of retinal ganglion cells (RGCs), cells that form the optic nerve • Precise mechanism of how RGCs die is not known • Currently no approved treatment for LHON patients • No clinical relevant model to study LHON disease

  9. Disease modelling Patient-specific iPS cells Retinal ganglion cells Study the disease mechanism: • Extremely difficult to obtain RGC samples from living patient to study LHON disease • Currently no RGC cell lines available • Potentials of iPS cells” • ‘Disease-in-a-dish’: understand how RGC loss occurs in LHON, which is crucial to developing new treatment

  10. Drug screening • Current cost of drug discovery is very expensive and takes a long time • 1.3-5 billion USD development cost, often takes >10 years • ~1-5% success rate for experimental drugs to make it to the clinic • No approved treatment in the case of LHON • Potentials of iPS cells: • Fast-track the process of drug discovery and testing with clinical relevant model • ‘Personalised medicine’

  11. Cell therapy • Immuno-rejection with transplantation • Cell therapy is especially helpful in diseases where affected cells are ‘damage beyond repair’ • The need for animal experiment before clinical trial in patients • Potential of iPS cells in LHON: • Replace damaged RGCs for restore vision • Unlimited source of cells • Autologous transplantation = minimal risk of graft rejection

  12. Acknowledgements: Neuroregeneration Unit, CERA Dr Alice Pebáy Ms Katie Gill Ms Grace Lidgerwood Dr Sandy Hung Dr Kathryn Davidson Ms Alison Conquest Ms Tejal Kulkarni Mr Duncan Crombie Ms Lerna Jurdukian FundingCranbourne Foundation Brookhoff Foundation Ophthalmic Research Institute of Australia University of Melbourne ECR grant Neuroregeneration Unit, 2013

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