University of Arkansas to Lead $1.4M Study on Rare Disease

October 20, 2016

The University of Arkansas will use a $1.4 million grant to study the origin of Leigh’s Disease, a rare and incurable disease that affects the central nervous system.

The study will last three years and the goal is to provide a better understanding of the emergence of the mitochondrial mutation that causes the disease, according to Shilpa Iyer, assistant professor of biological sciences at the U of A.

Iyer is leading the study.

“We are mimicking the earliest stages of human development to trace how the mitochondria mutation is transmitted in each cell,” Iyer said.”In essence, we are re-creating the disease in the lab. We are trying to provide hope for people who are very sick.”

If the study is successful, the researchers will generate clinical-grade, patient-specific stem cells for drug discovery and transplantation therapies for Leigh’s syndrome patients.


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