New U-M stem cell line looks to tackle Charcot-Marie-Tooth disease

The second stem cell line for the University of Michigan has made the U.S. National Institutes of Health’s registry. This makes the stem cell line research eligible for federal funding and makes U-M one of only four institutions to achieve this milestone.

Stem cells are known for their potential to show how genetic diseases start and develop. U-M's latest stem cell line carries the gene defect responsible for Charcot-Marie-Tooth disease, a hereditary neurological disorder characterized by a slowly progressive degeneration of the muscles in the foot, lower leg and hand. Charcot-Marie-Tooth disease is one of the most common inherited neurological disorders, affecting one in 2,500 people in the U.S.

"What we can do now is study as a cell differentiates," says Gary Smith, professor of OBGYN, physiology and urology at the University of Michigan. "You can begin to see exactly how the cells develop abnormally. We will be able to see it for the first time. When you see these cells develop abnormally yo can think of ways to circumvent it."

However, that process requires years of research before it can begin producing viable results to treat the disease. Smith estimates it will be 5-10 years before the fruits of this stem cell line research can be realized, if not longer.

"It will take time," Smith says. "It will be years. That is what good rigorous science does."

Source: Gary Smith, professor of OBGYN, physiology and urology at the University of Michigan
Writer: Jon Zemke

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