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National Glaucoma Research - Current Award

Raquel Lieberman

Raquel Lieberman, Ph.D.

Georgia Institute of Technology
Atlanta, GA

Title: Development of pharmacological chaperone therapy for inherited primary and juvenile open angle glaucoma
Non-Technical Title: The search for a new therapy for inherited glaucoma

Acknowledgements: Recipient of the Thomas R. Lee award for National Glaucoma Research
Duration: April 1, 2008 - March 31, 2011
Award Type: Standard
Award Amount: $100,000


Summary:

The researchers' aim is to develop a new therapy for inherited glaucoma, which in many cases is caused by mutations in a protein called myocilin.

Details:


The researchers' aim is to develop a new therapy for inherited glaucoma, which in many cases is caused by mutations in a protein called myocilin. Myocilin forms part of the trabecular extracellular matrix (TEM) in the eye and is important in regulating eye pressure. When the TEM doesn't function correctly, eye pressure increases, leading to retinal degeneration and vision loss. Human trabecular meshwork (HTM) cells, which produce the matrix, recognize mutations in myocilin and prevent mutant myocilin from being secreted to the TEM. Instead, mutant myocilin remains in the interior of HTM cells, causing the HTM cells to die. This disrupts the TEM, causing increased eye pressure and eventually glaucoma. The team hopes to discover a drug molecule that interacts with mutant myocilin inside the HTM cells to restore secretion of myocilin to the TEM. This could prevent the mutant protein from accumulating in the HTM cells and keep these cells alive. In addition, the TEM could be restored and better able to control eye pressure. This should slow the retinal degeneration associated with glaucoma. The researchers seek to better understand the molecular structure of myocilin, and then to identify and test drug candidates that bind to the myocilin.

Publications:

Burns, J. N., Orwig, S. D., Harris, J. L., Watkins, J. D., Vollrath, D. and Lieberman, R. L. (2010) Rescue of glaucoma-causing mutant myocilin thermal stability by chemical chaperones. ACS Chemical Biology, ASAP 3/24/10. (DOI: 10.1021/cb900282e) PubMed Icon Google Scholar Icon

The study reports the first high yield expression and purification of OLF, including a new assay to compare stability, and demonstration that 4 disease-causing mutants can be stabilized to near wild-type levels by small molecules. This publication was just released.