The Weizmann Institute of Science in Rechovot announced this week that researchers at the facility have the found the solution to the three-dimensional structure of an enzyme called glucocerebrosidase. Mutations occurring in this enzyme cause Gaucher disease, a genetic illness that mainly affects Ashkenazi Jews. The Institute study, published recently in EMBO Reports, 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, as well as Dr. Michal Harel, Lilly Toker and graduate student Hay Dvir, 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. Another type of therapy likely to emerge from the Weizmann findings is the design of small molecules that will supplement the damaged enzyme in the patient’s body, thereby restoring its normal functioning. Yeda Research and Development Co., which deals with the commercialization of Weizmann Institute research, has filed a patent application for the medical applications of these findings.

This entry was posted on Thursday, June 12th, 2003 and is filed under news.