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2 CELLS ARE THE FUNCTIONAL UNIT OF THE HUMAN BODY CELLS ARE THE FUNCTIONAL UNIT OF THE HUMAN BODY  SMALL BUSINESSES CELLS HAVE DIFFERENT JOBS CELLS HAVE DIFFERENT JOBS  SPECIALIZE/DIFFERENTIATED  PANCREATIC CELLS-INSULIN  NEURONS-TRANSMIT SIGNALS  HEART CELLS-CONTRACT TO PUMP BLOOD MANY DISEASES ARE CAUSED BY BREAKDOWNS IN THE CELL MANY DISEASES ARE CAUSED BY BREAKDOWNS IN THE CELL  SOMETIMES CELL ALIVE BUT DAMAGED  SOMETIMES CELL DIES AND IS LOST BASIC PRINCIPLES

3 MANY DISEASES ARE ASSOCIATED WITH CELL DAMAGE OR LOSS DIABETES-PANCREATIC CELLS DIABETES-PANCREATIC CELLS SPINAL CORD AND OTHER INJURIES SPINAL CORD AND OTHER INJURIES HEART FAILURE AND OTHER CARDIOVASCULAR DISEASE-HEART MUSCLE HEART FAILURE AND OTHER CARDIOVASCULAR DISEASE-HEART MUSCLE ALZHEIMER’S DISEASE-BRAIN CELLS ALZHEIMER’S DISEASE-BRAIN CELLS PARKINSON’S DISEASE-BRAIN CELLS PARKINSON’S DISEASE-BRAIN CELLS RETINITIS PIGMENTOSA-RETINAL CELLS RETINITIS PIGMENTOSA-RETINAL CELLS MUSCULAR DYSTROPHY-MUSCLE MUSCULAR DYSTROPHY-MUSCLE KIDNEY AND LIVER DISEASE KIDNEY AND LIVER DISEASE

4 BOTTLENECKS IN DISCOVERY OF NEW THERAPIES SOURCES OF CELLS FOR CELL AND TISSUE TRANSPLANTATION SOURCES OF CELLS FOR CELL AND TISSUE TRANSPLANTATION DEVELOPING NEW SAFE AND EFFECTIVE DRUGS DEVELOPING NEW SAFE AND EFFECTIVE DRUGS  HUMAN TOXICITY  HUMAN EFFICACY INCOMPLETE UNDERSTANDING OF DISEASE MECHANISMS INCOMPLETE UNDERSTANDING OF DISEASE MECHANISMS INDIVIDUAL PATTERNS OF DISEASE DEVELOPMENT AND DRUG RESPONSE INDIVIDUAL PATTERNS OF DISEASE DEVELOPMENT AND DRUG RESPONSE

5 HOW CAN STEM CELLS HELP? WHAT ARE STEM CELLS? WHAT ARE STEM CELLS?  CELLS THAT CAN DIVIDE TO GIVE RISE TO:  MORE STEM CELLS  CELLS THAT CAN ADOPT SPECIALIZED JOBS WHY ARE STEM CELLS PROMISING? WHY ARE STEM CELLS PROMISING?  THE RAW MATERIAL TO REPLACED DAMAGED CELLS AND ORGANS?  TOOLS TO UNDERSTAND AND COMBAT DISEASE  TOOLS TO TEST AND DEVELOP NEW DRUGS A NEW ENABLING TECHNOLOGY WITH BROAD APPLICATION? A NEW ENABLING TECHNOLOGY WITH BROAD APPLICATION?

6 HOW ARE PLURIPOTENT/EMBRYONIC STEM CELLS DERIVED? SPERM ZYGOTE (TOTIPOTENT) BLASTOCYSTFETUSFERTILIZATION INNER CELL MASS (PLURIPOTENT) EGG (OOCYTE) ADAPTED FROM ADULT IMPLANTATION INNER CELL MASS PRIMORDIAL GERM CELLS CULTURED PLURIPOTENT STEM CELLS EMBRYONIC STEM CELLS (ES) EMBRYONIC GERM CELLS (EG) IN THE LABORATORYIN THE UTERUS

7 EMBRYONIC STEM CELLS ARE PLURIPOTENT CULTURED PLURIPOTENT STEM CELLS

8 HUMAN NEURONS IN A DISH? DAPIB-tub III TH

9 WHAT DO WE KNOW ABOUT PLURIPOTENT/EMBRYONIC STEM CELLS? STRONG, RELIABLE, HIGHLY REPRODUCIBLE DATA ESTABLISH PLURIPOTENCY (ALMOST 20 YEARS OF DATA ON EXPERIMENTAL ORGANISMS) STRONG, RELIABLE, HIGHLY REPRODUCIBLE DATA ESTABLISH PLURIPOTENCY (ALMOST 20 YEARS OF DATA ON EXPERIMENTAL ORGANISMS) SOLID THEORETICAL AND EXPERIMENTAL FOUNDATION TO: SOLID THEORETICAL AND EXPERIMENTAL FOUNDATION TO:  INDUCE DEVELOPMENT OF SPECIALIZED CELL TYPES  SOLVE REJECTION PROBLEM CAN HAVE GOOD GROWTH PROPERTIES CAN HAVE GOOD GROWTH PROPERTIES PROMISING “PROOF OF PRINCIPLE” RESEARCH PROMISING “PROOF OF PRINCIPLE” RESEARCH

10 SOME EXISTING EXAMPLES OF THE USE OF PLURIPOTENT/EMBRYONIC STEM CELLS PARTIAL REPAIR OF SPINAL CORD DAMAGE, MULTIPLE SCLEROSIS, AND PARKINSON’S DISEASE MODELS IN RAT PARTIAL REPAIR OF SPINAL CORD DAMAGE, MULTIPLE SCLEROSIS, AND PARKINSON’S DISEASE MODELS IN RAT SHORT-TERM RELIEF OF PARKINSON’S DISEASE SYMPTOMS IN MONKEYS SHORT-TERM RELIEF OF PARKINSON’S DISEASE SYMPTOMS IN MONKEYS INDUCED DIFFERENTIATION TO MUSCLE, NEURONS, PANCREATIC CELLS, BLOOD-FORMING STEM CELLS INDUCED DIFFERENTIATION TO MUSCLE, NEURONS, PANCREATIC CELLS, BLOOD-FORMING STEM CELLS STUDIES OF DISEASE MECHANISM STUDIES OF DISEASE MECHANISM