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Why a hereditary anemia is caused by genetic mutation in mechanically sensitive ion channel
Date:3/8/2013

. The increased stress caused by this loss of reinforcement causes the channels to open and the leak of calcium is likely what causes the muscles to atrophy, Sachs explains.

Sachs and colleagues at UB founded a biotech company in Buffalo, Tonus Therapeutics to create a therapy for muscular dystrophy based on a peptide they discovered that inhibits the channels involved in that disease. They originally discovered the peptide in a tarantula venom but now it is synthesized chemically. The peptide has received orphan drug designation from the FDA.

"We were pleased to find that our spider venom peptide also inhibits the PIEZO1 channel," says Sachs.

"This means our peptide could be a potential therapy in blood diseases, where there are defects in the ways that red blood cells regulate cell volume," he says.

In normal cells, he says, the mechanosensitive ion channels usually remain closed.

"I think the cells use them as emergency valves so the only time they open is when cells are under extreme stress," he explains. "Consequently, our peptide doesn't bother healthy cells, so it's nontoxic. It only affects unhealthy cells, cells which are mechanically stressed."


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Contact: Ellen Goldbaum
goldbaum@buffalo.edu
716-645-4605
University at Buffalo
Source:Eurekalert

Page: 1 2

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