This document discusses using induced pluripotent stem cells (iPSCs) derived from a patient's own skin cells to generate dopamine-producing neurons for treating Parkinson's disease. Parkinson's involves the death of dopamine neurons in the brain. Previous research using embryonic stem cells in rodents led to tumor formation. The proposed methodology would involve taking skin cells from rodents, generating iPSCs, differentiating them into dopamine neurons, and surgically implanting them into the rodents' brains. This approach could reduce immunorejection risks and provide a new treatment for Parkinson's by replacing lost dopamine neurons. Future studies would assess the safety and effectiveness of this iPSC-based therapy.

1. PARKINSON DISEASE- SPECIFIC INDUCED
PLURIPOTENT STEM CELLS
Sara C. Díaz-Méndez
RISE Program
Enrique Rodriguez-Borrero, Ph.D

2. PARKINSON'S DISEASE (PD)

3. Parkinson's is a
progressive movement
disorder that usually strikes
after age 50.
Parkinson’s involves the
malfunction and death of
vital nerve cells in the brain,
called neurons.
The neurons that die in
Parkinson's Disease connect
a structure in the brain
called the substantia nigra to
another structure called the
striatum.
EFFECTS:

4. Alternative Approaches for
treating Parkinson's
disease:
• For many years, doctors have
treated Parkinson's disease patients
with the drug levodopa, which the
brain converts into dopamine.
Although the drug works well
initially, levodopa eventually loses its
effectiveness, and side-effects
increase.
 Surgery, such as deep brain
stimulation with implanted
electrodes, is used to treat
advanced symptoms. But it does not
slow down or reverse the damage to
nerve cells in the brain. Over time,
the symptoms often get worse
despite treatment.
 Pluripotent embryonic stem cells
have been recently used by
scientists and are under
investigation.

5. WHAT ARE PLURIPOTENT STEM CELLS?
 Stem cells are defined by two properties. First,
they can ‘self-renew,’ that is, they can divide and
give rise to more stem cells of the same kind.
Second, they can mature or ‘differentiate’ into
specialized cells that carry out a specific function.

6. PROBLEM
• Recent studies in rodents with embryonic stem
cells to replace dopamine producing neurons in
the brain have develop tumors as a side effect.

7. CHALLENGE
 Use a different type of pluripotent stem cells that won’t be
rejected from a person's body due to their immune system.
 Many scientists and members of the public have ethical issues
with the use of pluripotent stem cells from human embryos or fetal
tissues.

8. HYPOTHESIS
Using pluripotent cells from the patient’s skin to produce pluripotent
embryonic like cells and from these cells generate the dopamine
producing neurons that will reduce the risk of immunological
rejection of the new neuron cells.

9. METHODOLOGY
1. I will use a large number of rodents that will allow my results to be
statistically significant. First I will locate the midbrain area in rodents
involved in dopamine production.
1. Induced pluripotent stem (iPS) cells are going to be made from a
rodent’s skin cells in the lab, and then used to make dopamine-
producing neurons.
- To make a specific type of cell in high numbers, scientists expose the
stem cells to a cocktail of chemicals that mimic what they would
experience during normal development. The resulting cells also carry
more of the molecular markers that characterize dopamine-producing
cells in the brain.

10. METHODOLOGY
• 3. Surgically I will introduced the generated dopamine producing
neurons in the midbrain area, which is where dopamine neurons
are normally located. Since a single, well-defined type of cell is
affected, it is possible to treat Parkinson’s by replacing the lost
nerve cells with healthy new ones.
• 4. Their physical and motor skills will be observe to evaluate the
progress of the disease. Laboratory blood analysis will be
performed to monitor the progress of the disease.

11. FUTURE INVESTIGATION
• Studies will be needed to make sure the cells are safe and
would not cause tumors in the brain.

12. EXPECTED OUTCOME
• This will improve the quality of life of elderly people affected by
Parkinson's Disease and extend their life expectancy. It will also
reduce the cost of caring of elderly people.

13. REFERENCES:
• "All About Parkinso's Disease." . Marc, 25 Apr. 2014. Web. 21 June 2014.
<http://coloradodementia.org/category/parkinsons-disease-2/>.
• "Parkinson's Disease." . NIH Senior Health, 1 June 2012. Web. 1 June 2014.
<http://nihseniorhealth.gov/parkinsonsdisease/whatcausesparkinsonsdisease/01.html>.
• "STEM CELL RESEARCH AND PARKINSON’S DISEASE." . Parkinson Society Canada, 1
May 2009. Web. . <http://www.parkinson.ca/atf/cf/%7B9EBD08A9-7886-4B2D-A1C4-
A131E7096BF8%7D/stem%20cells%20-%20en.pdf>.
• "Reprogramming: how to turn any cell of the body into pluripotent stem cell ." . Euro Stem
Cell , 14 Dec. 2012. Web. . <http://www.eurostemcell.org/factsheet/reprogramming-how-
turn-any-cell-body-pluripotent-stem-cell>.
• "Stem Cells and Diseases." . National Institude of Health, 1 Dec. 2013. Web. .
<http://stemcells.nih.gov/info/pages/health.aspx>.