Therapeutic Possibilities of

Stem Cell Research

Cystic Fibrosis: Stem Cell-
Gene 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

“Blueprint” cells
Inner Cell Mass
(Stem Cells)

A Blastocyst is a hollow ball of cells

with a small clump of stem cells inside
 Human Embryonic Stem Cells
Blastocyst ­
from In Vitro Fertilization Clinic

Pipette

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
 Human Embryonic Stem Cells
Blastocyst ­
from In Vitro Fertilization Clinic Pipette

“Blueprint” 
Stem Cells
cells
Stem Cells “Blueprint” cells Stem Cells

A Blastocyst is a hollow ball of cells with a  To remove the stem cells, the Blastocyst is 
small clump of stem cells inside  broken open and the stem cells removed with 
a pipette(an ultra thin glass tube)

The stem cells are

placed in a Pipette
dish and are fed and
cared for
(each blastocyst =
1 stem cell line) Petri Dish
 Growth factors Chemical cues

Petri Dish
Stem Cells

Pancreatic 
Islet  Muscle
Neuron cell

Different chemicals / molecules are added to the stem 
cells to make them become specific types of cells.  
 Somatic Cell Nuclear Transfer or Therapeutic Cloning
Donor Egg Skin Cell
Nucleus Nucleus
(DNA) (DNA)

Needle Needle

Stem Cells
Chemicals and 
Growth Factors

Pancreatic 
Islet

Blastocyst  Neuron
Stem Cells
Dividing cells 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
• Brain and spinal cord injury and stroke (loss
of nerve cells and myelin-forming
oligodendrocytes).

• 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).
 Goal #1: to make stem cells into nerve cells Stem 
Blastocyst ­
Pipette Cells

“Blueprint” 
cells

Petri Dish
Stem Cells “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 Then to

differentiated to the Oligodendrocyte
neural lineage. 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 Heart
Fat
Bone marrow Cartilage