UW-Madison researchers have found a new way to potentially treat Friedreich’s ataxia, a rare, fatal and currently untreatable disorder.
Aseem Ansari, a professor of biochemistry and genomics at UW-Madison and leader of the research team that made this discovery, says this method represent a “new precision-tailored path to personalized medicine.”
Friedreich’s ataxia afflicts 1 in every 50,000 people, according to Ansari. That’s equal to about 115 cases in Wisconsin.
The disorder is caused by stretches of repetitive DNA in an individual’s genetic code which prevent the creation of an important energy-processing protein.
“These kids accumulate repeats in a gene for a protein called frataxin that mitochondria, the cell’s powerhouse, need to process energy. Without frataxin, tissues that use the most energy get hurt first: the brain, heart and pancreas,” Ansari says.
According to Froedtert and the Medical College of Wisconsin, a wide array of symptoms like tiredness, slurred speech, difficulty walking, loss of key senses like vision and hearing, and others can occur between ages 5 and 15, though some do crop up later in life.
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