Imagine losing the ability to control your physical movements or the ability to control coordination. These two symptoms, along with many more, happen to people who suffer from the genetically dominant, neurodegenerative disorder Huntington's Disease. Currently, there is no cure for Huntington's Disease and no way to stop the disease once it has begun. Even though there are a few drugs available to relieve some of the symptoms that occur with the disease, researchers are actively looking for treatment options.
Recently, researchers at Washington University School of Medicine in St. Louis developed a method to replace some brain cells that are affected by Huntington's Disease. (Image courtesy of Allan Ajifo via Wikimedia Commons).
The researchers at Washington University St. Louis found that they could reprogram cells from adult human skin to be identical to a specific type of brain cell that helps control movement. The brain cells that they replicated, called Medium Spiny Neurons, are the main cells affected by Huntington's Disease. Once the researchers reprogrammed the human skin cells, they inserted them into mice and saw that the new cells behaved very similarly to the native Medium Spiny Neuron cells already in the brain.
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"Not only did these transplanted cells survive in the mouse brain, they showed functional properties similar to those of native cells,” said researcher and assistant professor of developmental biology Andrew S. Yoo, Ph.D. “These cells are known to extend projections into certain brain regions. And we found the human transplanted cells also connected to these distant targets in the mouse brain."
The process of taking one type of cell and reprogramming it to behave like a different type of cell has been done in previous research studies. The research team on this study knew that two small microRNA molecules had the ability to change the DNA of human skin cells into different types of neurons, in this case, Medium Spiny Neurons found in the brain. How these microRNA molecules actually alter the DNA is still being studied, but the researchers knew that by altering chemical signals they could force the skin cells to be exposed to transcription factor molecules near the brain cells, responsible for changing cells from one type to another.
Matheus B. Victor, a neuroscience graduate student and part of this WUSTL research team, explained this process by the microRNA molecules: “They are priming the skin cells to become neurons. The transcription factors we add then guide the skin cells to become a specific subtype, in this case medium spiny neurons."
Now that the researchers know that the newly programmed cells will live in the mice brain and function closely to original brain cells, they plan to test this process by using human skin cells taken directly from patients suffering from Huntington's Disease and using them in mice to see if this has any effect on the symptoms of Huntington's Disease. If this process works and the skin cells can replace affected brain cells, there is the possibility that this method can be used to further treat Huntington's Disease symptoms.
Washington University. (Image courtesy of Biotechnology Calendar, Inc.)
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