Inhibition of endoplasmic reticulum-associated degradation rescues native folding in loss of function protein misfolding diseases.

@article{Wang2011InhibitionOE,
  title={Inhibition of endoplasmic reticulum-associated degradation rescues native folding in loss of function protein misfolding diseases.},
  author={Fan Wang and Wensi Song and Giovanna Brancati and Laura Segatori},
  journal={The Journal of biological chemistry},
  year={2011},
  volume={286 50},
  pages={43454-64}
}
Lysosomal storage disorders are often caused by mutations that destabilize native folding and impair trafficking of secretory proteins. We demonstrate that endoplasmic reticulum (ER)-associated degradation (ERAD) prevents native folding of mutated lysosomal enzymes in patient-derived fibroblasts from two clinically distinct lysosomal storage disorders, namely Gaucher and Tay-Sachs disease. Prolonging ER retention via ERAD inhibition enhanced folding, trafficking, and activity of these unstable… CONTINUE READING

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ERAD Inhibition Facilitates Mutant Enzyme

J. Lu, J. Chiang, +9 authors Z. andZhuang
Folding DECEMBER • 2011

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