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Scripps research scientists shed light on potential treatment for Gaucher's disease
Date:5/9/2010

(SERCA) on endoplasmic reticulum calcium levels," said Ong. "We found that one of the calcium efflux channels was sufficient to increase calcium levelsthe ryanodine receptor."

These drugs appear to enhance the function of a chaperone called calnexin, which is expected to aid the folding of most lysosomal enzymes because of their common biochemistry (post-translational modificationN-linked glycosylation).

While Ong is hopeful that the drugs will be effective for treating Gaucher's disease and other lysosomal storage disorders, he also cautions that many steps still need to be taken before such treatment is approved for use in patients.

"We are always cautious because what works in patient-derived cells does not always work in humans," he said.


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Contact: Keith McKeown
kmckeown@scripps.edu
858-784-8134
Scripps Research Institute
Source:Eurekalert

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