Stem Cells and Regenerative Medicine Zach Scheiner Ph

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Stem Cells and Regenerative Medicine Zach Scheiner, Ph. D Science Officer

Stem Cells and Regenerative Medicine Zach Scheiner, Ph. D Science Officer

What are stem cells? What are the different types of stem cells? Why should

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?

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

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

Self-Renewal (symmetric cell division)

Self-Renewal (symmetric cell division)

Differentiation (asymmetric cell division) Progenitor cell Stem cell

Differentiation (asymmetric cell division) Progenitor cell Stem cell

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

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

Stem Cell Types • Embryonic – pluripotent: can form almost any cell type in

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

In Conception Vitro Fertilization in a Dish

In Conception Vitro Fertilization in a Dish

In Vitro Fertilization Day 1

In Vitro Fertilization Day 1

In Vitro Development Day 1

In Vitro Development Day 1

In Vitro Development Day 2

In Vitro Development Day 2

In Vitro Development Day 2

In Vitro Development Day 2

In Vitro Development Day 3

In Vitro Development Day 3

In Vitro Development Day 4

In Vitro Development Day 4

In Vitro Development Day 5 Embryonic Stem Cells

In Vitro Development Day 5 Embryonic Stem Cells

This cell can form all of the cells in the human body This cell

This cell can form all of the cells in the human body This cell can form almost every cell type in the human body Totipotent Pluripotent Fully mature

Human embryonic stem cells in culture

Human embryonic stem cells in culture

Fluorescent imaging of human embryonic stem cell colonies

Fluorescent imaging of human embryonic stem cell colonies

What Diseases Do Stem Cells Treat? Have the Potential to Treat? Currently Treat Potential

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

Bone Marrow (Hematopoietic Stem Cell) Transplant Example of a tissue-specific stem cell therapy

Bone Marrow (Hematopoietic Stem Cell) Transplant Example of a tissue-specific stem cell therapy

Trachea transplantation Example of tissue-specific stem cell-based tissue replacement Click on Picture to Play

Trachea transplantation Example of tissue-specific stem cell-based tissue replacement Click on Picture to Play Video

Tissue-specific (adult) stem cells are powerful and promising! Why do researchers study embryonic stem

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!

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

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

Applications for Embryonic Stem Cells Video: Diabetes A CIRM Disease Team Video: Age-Related Macular

Applications for Embryonic Stem Cells Video: Diabetes A CIRM Disease Team Video: Age-Related Macular Degeneration (AMD) A CIRM Disease Team

Video: CIRM Disease Team for Type 1 Diabetes

Video: CIRM Disease Team for Type 1 Diabetes

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

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

What are some of the challenges facing embryonic stem cell research? • Differentiation of

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

Induced Pluripotent Stem (i. PS) Cells Genetically engineering new stem cells Virus engineered to

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

Video: Parkinson’s“disease-in-a-dish”

Video: Parkinson’s“disease-in-a-dish”

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

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

Extra Slides

Extra Slides

Neural stem cells for drug delivery Focused delivery of chemotherapy for cancer Day 0

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

Stem cells for drug delivery Focused delivery of chemotherapy for cancer Another CIRM Disease

Stem cells for drug delivery Focused delivery of chemotherapy for cancer Another CIRM Disease Team Genetically Engineered Neural Stem Cells Mice are given a nontoxic drug, which the neural stem cells can convert to an active drug to shrink tumors!

Milestones in Embryonic Development • Days 7 -14: Embryo implants in the uterus •

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