PARKINSON DISEASE- SPECIFIC INDUCED

PLURIPOTENT STEM CELLS

Sara C. Díaz-Méndez

RISE Program

Enrique Rodriguez-Borrero, Ph.D

PARKINSON'S DISEASE (PD)

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:

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.

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.

PROBLEM

• Recent studies in rodents with embryonic stem

cells to replace dopamine producing neurons in

the brain have develop tumors as a side effect.

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.

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.

METHODOLOGY1. 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 rodentsinvolved in dopamine production.

1. Induced pluripotent stem (iPS) cells are going to be made from arodent’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 thestem cells to a cocktail of chemicals that mimic what they wouldexperience during normal development. The resulting cells also carrymore of the molecular markers that characterize dopamine-producingcells in the brain.

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.

FUTURE INVESTIGATION

• Studies will be needed to make sure the cells are safe and

would not cause tumors in the brain.

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.

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>.