Therapeutic Possibilities of Stem Cell Research

Cystic Fibrosis: Stem CellGene Therapy Approach:
• n Human bone marrow
derived stem cells can differentiate into airway epithelial cells n Encoding these cells with the gene that is defective in CF restores cellular function n Keep airways clear of mucus and air-borne irritants n Hope to perform clinical trial in next 2-3 years

• •

Biological Pacemaker:
• n Human Embryonic • • • •
Stem Cells genetically engineered and coaxed to become heart cells n Clusters of cells beat on their own triggered the unified beating of rat heart muscle cells n Triggered regular beating when implanted in guinea pigs n Cells responded to drugs used to slow or speed up heart rate n Use genetic engineering to customize the pacing rate of the cells

Retinal Degeneration:
• n Mice predisposed for • • • •
Retinitis Pigmentosa: a degenerative disease that destroys retinas n Injected bone marrowderived stem cells into the back of mouse eyes during development n Dramatically curtailed retinal degeneration n Completely normal vasculature, improved retinal tissue and light response n Disorders of the retina that have vascular and neuronal degeneration: genetic disorders known collectively as retinitis pigmentosa

A primer on Human Embryonic Stem Cells Blastocyst from In Vitro Fertilization Clinic

Inner Cell Mass (Stem Cells)

“Blueprint” cells

A Blastocyst is a hollow ball of cells with a small clump of stem cells inside

Blastocyst ­

Human Embryonic Stem Cells
Pipette

from In Vitro Fertilization Clinic

Stem Cells

“Blueprint” cells

A Blastocyst is a hollow ball of cells with a  small clump of stem cells inside 

Stem Cells

“Blueprint” cells

To remove the stem cells, the Blastocyst is opened and the stem cells removed with a pipette

Blastocyst ­

Human Embryonic Stem Cells
Pipette

from In Vitro Fertilization Clinic

Stem Cells

“Blueprint” cells

Stem Cells

“Blueprint”  cells

Stem Cells

A Blastocyst is a hollow ball of cells with a  small clump of stem cells inside 

To remove the stem cells, the Blastocyst is  broken open and the stem cells removed with  a pipette(an ultra thin glass tube)

The stem cells are placed in a dish and are fed and cared for (each blastocyst = 1 stem cell line)

Pipette

Petri Dish

Growth factors

Chemical cues

Stem Cells

Petri Dish

Pancreatic  Islet 

Neuron

Muscle cell

Different chemicals / molecules are added to the stem  cells to make them become specific types of cells.  

Donor Egg

Somatic Cell Nuclear Transfer or Therapeutic Cloning
Skin Cell Nucleus (DNA) Needle Nucleus (DNA) Needle

Chemicals and  Growth Factors

Stem Cells

Pancreatic  Islet Neuron

Dividing cells

Stem Cells

Blastocyst 

Muscle cell

The vision for UCI’s Stem Cell Center: Stem cell therapies for neurological disorders

• Brain and spinal cord
injury.

• Stroke. • Neurodegenerative
diseases
– – – – Parkinson’s Disease Huntington’s Disease Alzheimer’s Disease Multiple Sclerosis

Neurological disorders involve the loss of particular cell types in the nervous system
of nerve cells and myelin-forming oligodendrocytes).

• Brain and spinal cord injury and stroke (loss

• Neurodegenerative diseases

– Parkinson’s Disease (loss of dopamine-containing nerve cells in the brainstem). – Huntington’s Disease (loss of nerve cells in the striatum). – Alzheimer’s Disease (loss of nerve cells in the cerebral cortex). – Multiple Sclerosis (loss of myelin-forming oligodendrocytes). – Lou Gerhig’s Disease-ALS (loss of motor neurons from the spinal cord).

Blastocyst ­

Goal #1: to make stem cells into nerve cells
Pipette “Blueprint”  cells

Stem  Cells

Stem Cells

Petri Dish
“Blueprint” cells Stem Cells

The stem cells are treated with  factors to cause them to differentiate  into particular cell types

Stem cells differentiated into neurons

Goal #2: To discover how to make stem cells  integrate into neural circuits.
Nerve cell (neuron) Oligodendrocyte

More research is needed to find the  ways to actually use stem cells for  therapeutic applications.

At the RIRC, a therapy is being developed to use stem cells to replace myelin-forming cells
Myelin­forming cells (oligodendrocytes) die  as a result of spinal cord injury, resulting in  the loss of myelin (insulation) from nerve  fibers. An important potential therapeutic strategy:   Replace myelin­forming cells using stem  cells that differentiate into oligodendrocytes.

Reeve-Irvine Research Center

RIRC scientists have succeeded in developing ways to produce  oligodendrocytes from human ES cells and have shown that they  can restore myelin after spinal cord injury in experimental  animals.

Stem cells are first differentiated to the neural lineage.

Then to Oligodendrocyte Precursors

But do these cells have the potential to form tumors over longer periods of time?

Embryonic Stem Cells
1. They can generate large quantities of tissue rapidly

Human Embryonic Stem Cells

2. They can become any cell in the body
Embryonic stem cells

Brain Bone marrow Fat Cartilage

Heart

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