Gaucher Disease Code Cracked

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The Weizmann Institute of Science in Rehovot announced last week that its researchers had found the solution to the three-dimensional structure of an enzyme called glucocerebrosidase. Mutations occurring in this enzyme cause Gaucher's Disease, a genetic illness that mainly affects Ashkenazi Jews. The study may lead to the design of effective new therapies for treating the disease.

The Institute team, including Prof. Tony Futerman of the Biological Chemistry Department, Prof. Joel Sussman of the Structural Biology Department and Prof. Israel Silman of the Neurobiology Department, succeeded in growing the enzyme crystals by cutting parts of certain sugar molecules on the surface of the enzyme. The structural information gleaned by the Israeli team may help design a more effective enzyme that will improve today's therapy for Gaucher's Disease, until the development of gene therapy for this disorder is developed. Yeda Research and Development Company, which deals with the commercialization of Weizmann Institute research, has filed a patent application for the medical applications of these findings.






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