Updates

Duncan NRI Collaborative Research Programs

Telethon Institute of Genetics and Medicine (TIGEM)

Telethon Institute of Genetics and Medicine (TIGEM) was founded in 1994 by the Telethon Foundation, one of Italy's leading non-profit organizations, to promote research aimed at diagnosing, preventing and curing human genetic diseases. TIGEM invited world-renowned geneticist Dr. Andrea Ballabio, then on faculty at Baylor College of Medicine, to lead the new endeavor. The longstanding relationships between faculty at BCM and TIGEM underpin the new, formal collaboration centered at the Duncan NRI. 

The complementary expertise enjoyed by Duncan NRI and TIGEM in lysosomal biology, molecular genetics, cell biology, biochemistry, physiology, bioinformatics, systems biology and viral-mediated gene delivery will allow the collaborators to utilize a variety of animal models (mouse and fruit flies) of human diseases to test discoveries in living organisms before proceeding to clinical trials. Close interactions, the free exchange of data and ideas between researchers, and collaborative funding will accelerate progress in developing innovative therapies. The preliminary results in animal models of lysosomal storage disorders are already proving very promising.

Dr. Ballabio is widely acknowledged to be a world leader in the elucidation of the mechanisms of human genetic diseases. Dr. Ballabio's group identified the "master gene," TFEB, which regulates the expression of the CLEAR network in lysosomes. His group has shown that overexpression of TFEB in cultured cells and in murine models enhances lysosomal biogenesis, activates autophagy, and induces lysosomal exocytosis, all of which results in the efficient clearance of glycosaminoglycans, known to accumulate in lysosomal storage diseases, as well as toxic substrates such as the protein responsible for Huntington's disease. The next steps are to develop tools to modulate cellular clearance by acting on the CLEAR network and to use them to treat human diseases. The TIGEM-NRI program will be instrumental in meeting these challenges.

The Center for Drug Discovery 

The mission of the Center for Drug Discovery (CDD) located at the Duncan NRI is to develop small molecule probes, preclinical candidates, and drugs for researchers and clinicians in the Texas Medical Center and beyond. It is a resource for investigators in all Baylor College of Medicine departments and will complement efforts in the Department of Pharmacology, the Gulf Coast Consortia, and the Institute for Clinical and Translational Research as well as other departments and centers at Baylor College of Medicine. Led by Dr. Martin Matzuk and the assistant director, Dr. Damian Young, the CDD aims to bridge the gap between academic research and pharmaceutical discovery and provide researchers with an economical path to preclinical drug discovery.

The complementary expertise enjoyed by Duncan NRI and TIGEM in lysosomal biology, molecular genetics, cell biology, biochemistry, physiology, bioinformatics, systems biology and viral-mediated gene delivery will allow the collaborators to utilize a variety of animal models (mouse and fruit flies) of human diseases to test discoveries in living organisms before proceeding to clinical trials. Close interactions, the free exchange of data and ideas between researchers, and collaborative funding will accelerate progress in developing innovative therapies. The preliminary results in animal models of lysosomal storage disorders are already proving very promising.

Dr. Ballabio is widely acknowledged to be a world leader in the elucidation of the mechanisms of human genetic diseases. Dr. Ballabio's group identified the "master gene," TFEB, which regulates the expression of the CLEAR network in lysosomes. His group has shown that overexpression of TFEB in cultured cells and in murine models enhances lysosomal biogenesis, activates autophagy, and induces lysosomal exocytosis, all of which results in the efficient clearance of glycosaminoglycans, known to accumulate in lysosomal storage diseases, as well as toxic substrates such as the protein responsible for Huntington's disease. The next steps are to develop tools to modulate cellular clearance by acting on the CLEAR network and to use them to treat human diseases. The TIGEM-NRI program will be instrumental in meeting these challenges.

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