Stem Cells and Regenerative Medicine

Zach Scheiner, PhDScience 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?

All Stem Cells Can…

1. Self-Renew

2. Differentiate

Self-Renewal (symmetric cell division)

Differentiation (asymmetric cell division)

Progenitor cell

Stem cell Stem cell

Skin Progenitor Cell

Skin Cell

Neural Progenitor Cell

Neuron (Brain Cell)

Stem CellEmbryonic

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

Conception in a DishIn Vitro Fertilization

Day 1

In Vitro Fertilization

Day 1

In Vitro Development

Day 2

In Vitro Development

Day 2

In Vitro Development

Day 3

In Vitro Development

Day 4

In Vitro Development

EmbryonicStemCells

Day 5

In Vitro Development

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

This cell can formalmost every cell type

in the human body

Fully mature

Human embryonic stem cells in culture

Fluorescent imaging of human embryonic stem cell colonies

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)

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!

Currently Treat- Blood Diseases (including immune system disorders)- Genetic metabolic disorders (very limited/experimental)- Tissue/organ replacement (very limited/experimental)

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

Trachea transplantationExample 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 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

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 Age-Related Macular Degeneration (AMD) – Eye Disease

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 (iPS) CellsGenetically engineering new stem cells

Skin cells iPS cells

Pros: No embryos required No immune rejection? Disease in a dish?

Cons: May not be = to ESCs Genetically engineered

Virus engineeredto express fourkey “pluripotency”genes

Video: Parkinson’s“disease-in-a-dish”

Acknowledgements- Todd Dubnicoff- Amy Adams- Laurel Barchas

www.cirm.ca.gov

Extra Slides

Neural stem cells for drug deliveryFocused delivery of chemotherapy for cancer

NSCs injected

(no tumor)

NSCs injected (tumor)

Day 0 Day 7 Day 14

Shah et al. Dev Neurosci 2004

Stem cells for drug deliveryFocused delivery of chemotherapy for cancer

Another CIRM Disease Team

Genetically EngineeredNeural Stem Cells

Mice are given a non-toxic 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• 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