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Stem Cells and Regenerative Medicine

Stem Cells and Regenerative Medicine. Zach Scheiner, PhD Science Officer. What are stem cells? What are the different types of stem cells? Why should we study stem cells? What potential therapies could they provide? What are some of the challenges facing stem cell research?.

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Stem Cells and Regenerative Medicine

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  1. Stem Cells and Regenerative Medicine Zach Scheiner, PhD Science Officer

  2. What are stem cells? What are the different types of stem cells? Why should we study stem cells? What potential therapies could they provide? What are some of the challenges facing stem cell research?

  3. All Stem Cells Can… 1. Self-Renew 2. Differentiate

  4. Self-Renewal (symmetric cell division)

  5. Differentiation (asymmetric cell division) Progenitor cell Stem cell Stem cell

  6. Skin Cell Neuron (Brain Cell) Skin Progenitor Cell Neural Progenitor Cell Embryonic Stem Cell

  7. Stem Cell Types • Embryonic – pluripotent: can form • almost any cell type in the human body • Tissue-Specific (Adult) – • multipotent: can form only limited • types of cells (blood, brain, liver, etc.) • Induced Pluripotent – engineered • by scientists to act like embryonic stem cells

  8. In Vitro Fertilization Conception in a Dish

  9. In Vitro Fertilization Day 1

  10. In Vitro Development Day 1

  11. In Vitro Development Day 2

  12. In Vitro Development Day 2

  13. In Vitro Development Day 3

  14. In Vitro Development Day 4

  15. In Vitro Development Day 5 Embryonic Stem Cells

  16. Totipotent This cell can form all of the cells in the human body Pluripotent This cell can form almost every cell type in the human body Multi- potent Fully mature

  17. Human embryonic stem cells in culture

  18. Fluorescent imaging of human embryonic stem cell colonies

  19. What Diseases Do Stem Cells Treat? Have the Potential to Treat? • Currently Treat • Blood Diseases (including immune system disorders) • Genetic metabolic disorders (very limited/experimental) • Tissue/organ replacement (very limited/experimental) • Currently Treat • Blood Diseases (including immune system disorders) • Genetic metabolic disorders (very limited/experimental) • Tissue/organ replacement (very limited/experimental) • Potential to Treat • Heart Disease • Neurological Diseases (Parkinson’s, Alzheimer’s, Huntington’s & others) • Stroke • Type 1 Diabetes • Macular Degeneration (a common cause of blindness) • Cancer • HIV/AIDS • Spinal Cord Injury • Multiple Sclerosis • ALS (Lou Gehrig’s Disease) • Liver Disease • Potential to Treat • Heart Disease • Neurological Diseases (Parkinson’s, Alzheimer’s, Huntington’s & others) • Stroke • Type 1 Diabetes • Macular Degeneration (a common cause of blindness) • Cancer • HIV/AIDS • Spinal Cord Injury • Multiple Sclerosis • ALS (Lou Gehrig’s Disease) • Liver Disease …and more!

  20. Bone Marrow (Hematopoietic Stem Cell) TransplantExample of a tissue-specific stem cell therapy

  21. Trachea transplantationExample of tissue-specific stem cell-based tissue replacement Click on Picture to Play Video

  22. Tissue-specific (adult) stem cells are powerful and promising! • Why do researchers study embryonic stem cells? • Tissue-specific stem cells are limited in their differentiation potential (blood  blood) • Stem cells from some tissues are inaccessible • Some tissue-specific stem cells don’t self-renew well • Some tissues may not have stem cells!

  23. Embryonic Stem-Cell Derived Heart Muscle Cells Click on Pictures to Play Videos

  24. Applications for Embryonic Stem CellsVideo: Diabetes A CIRM Disease TeamVideo: Age-Related Macular Degeneration (AMD)A CIRM Disease Team

  25. Video: CIRM Disease Team for Type 1 Diabetes

  26. Video: CIRM Disease Team for Age-Related Macular Degeneration (AMD) – Eye Disease

  27. What are some of the challenges facing embryonic stem cell research? • Differentiation of stem cells into • mature, functional cells • Potential for tumor formation • Immune rejection

  28. Induced Pluripotent Stem (iPS) CellsGenetically engineering new stem cells Virus engineered to express four key “pluripotency” genes  Pros: No embryos required No immune rejection? Disease in a dish? Cons: May not be = to ESCs Genetically engineered Skin cells iPS cells

  29. Video: Parkinson’s“disease-in-a-dish”

  30. Acknowledgements • Todd Dubnicoff • Amy Adams • Laurel Barchas www.cirm.ca.gov

  31. Extra Slides

  32. Neural stem cells for drug deliveryFocused delivery of chemotherapy for cancer Day 0 Day 7 Day 14 NSCs injected (no tumor) NSCs injected (tumor) Shah et al. Dev Neurosci 2004

  33. Stem cells for drug deliveryFocused delivery of chemotherapy for cancerAnother CIRM Disease Team Genetically Engineered Neural Stem Cells Mice are given a non-toxic drug, which the neural stem cells can convert to an active drug to shrink tumors!

  34. Milestones in Embryonic Development • Days 7-14: Embryo implants in the uterus • Day 14: Three distinct layers begin to form (no more embryonic stem cells) • Days 14-21: Beginning of future nervous system • Days 21-24: Beginning of future head, neck, mouth, and nose • Weeks 3-8: Beginning of organ formation • Week 8: Embryo is called a fetus

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