Researchers at Fox Chase Cancer Center have demonstrated that it might be possible to treat genetic diseases, including some forms of cancer, by "rescuing" the misshapen, useless proteins produced by some mutant genes.

In the current issue of the Journal of Biological Chemistry, available online now, researchers detail how they were able to restore the function of a mutant human gene in a yeast model of disease by manipulating the available amounts of a so-called chaperone protein, named Hsp70, which helps amino acid chains fold into their proper protein form.

"In some cases, despite a mutation, it is possible to coax a misfolded protein back into a functional conformation," says the paper's lead author Warren Kruger, Ph.D., senior faculty member at Fox Chase. "In essence, we're using Hsp70 to call a biochemical mulligan, a do-over."

"Hsp70 pulls a misfolded amino acid chain apart like a twisted rubber band and allows it to snap back into place, which we found can restore a significant percentage of proteins to working shape," Kruger says. "If this can be done in humans, it could represent a way of reducing the severity - or perhaps correcting - certain hereditary diseases, even some familial cancers…