How scorpion stings could help CF patients

Article

The venom lying in wait in a scorpion's tail is certainly bad news to its prey. But it could contain the key to an effective cystic fibrosis (CF) treatment.

The venom lying in wait in a scorpion's tail is certainly bad news to its prey. But it could contain the key to an effective cystic fibrosis (CF) treatment.

For patients with CF, chaperone proteins are well-intentioned crusaders who do more harm than good. When they discard the bad CFTR (cystic fibrosis transmembrane conductance regulator) proteins, they stop flow of water to the lungs and airway.

How can an animal's poison can be used to help treat this? Scorpion venom, like snake and spider venom, is a peptide toxin. These work by blocking muscle ion or nerve channels, creating instant paralysis.

Researchers at Georgia Institute of Technology, working with other research groups, isolated a novel peptide from the venom of the Giant Israeli scorpion. This peptide, GaTx1, binds to the flawed CFTR proteins in the lab. If it were to bind to the same sites in a person with CF, the chaperone proteins would be unable to bind and discard them. This could mean more water flow to the lungs, looser mucus, and potentially less blocked airways.

The results were published in the Journal of Biological Chemistry.

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