Text Size Normal Text Sizing Button Medium Text Sizing Button Large Text Sizing Button Text Contrast Normal Contrast Button Reverse Contrast Button Switch to Spanish Language Press Room Contact Us Sitemap Sign In Register
Link to Homepage About BrightFocus
Donate Now Get Involved  
Alzheimer's Disease Research Macular Degeneration Research National Glaucoma Research

Sign up for Email Notifications
If you would like to be notified when funding or meeting opportunities are announced please click on the link below.

Sign up for new announcements.

Please add ResearchGrants@BrightFocus.org to your institution’s white list to insure that the notification is not blocked by your organization’s SPAM filters.

This email list is not sold or distributed, and serves only as an annual reminder of the availability of research support through the BrightFocus Foundation (www.brightfocus.org). Please follow instructions on the notification emails for removal requests.

BrightFocus Research Grants Funding
Grant Funding for Alzheimer's Research
Grant Funding for Macular Degeneration Research
Grant Funding for Glaucoma Research


Macular Degeneration Research
Completed Award

Photo Pending

Jayakrishna Ambati, M.D.

University of Kentucky Research Foundation
Lexington, KY

Title: Adeno-associated viral gene therapy in a novel mouse model of AMD
Non-Technical Title: Gene therapy for AMD

Duration: April 1, 2004 - June 30, 2008
Award Type: Standard
Award Amount: $100,000


This project uses viral gene therapy in mice to repair genetic defects that lead to AMD like pathology.


Progress in understanding how AMD develops and the process of developing new drug treatments has been hampered by the absence of good animal models. However, Dr. Ambati’s past research has shown that mice with a deletion of their Ccl-2 gene develop a form of ocular pathology similar to human AMD as they age. The development of early (dry) AMD as well as late (wet) AMD in these mice makes this model particularly attractive for scientists investigating the development of the disease and the mechanisms that mediate the progression of dry AMD to the more severe wet form. Dr. Ambati’s goal is to replace the function of the deleted Ccl-2 gene in these mice using an adeno-associated virus (AAV) as a vector. By treating one eye with active AAV therapy (which will deliver the Ccl-2 gene) and treating the other eye with a control AAV treatment, Dr. Ambati hopes to gain a better understanding of the molecular mechanisms underlying the development of AMD. These studies could also provide definitive proof that the absence of the Ccl-2 gene is directly responsible for the development of AMD in mice, and will justify an investigation of potential mutations in the Ccl-2 gene in humans with AMD. This in turn could lead to the identification of new therapeutic strategies to prevent blindness and restore sight.


Nozaki, M, Raisler, B.J., Sakurai, E, Sarma, J.V., Barnum, S.R., Lambris, J.D., Chen, Y, Zhang, K, Ambati, B.K., Baffi, J.Z., and Ambati, J. (2006) Drusen complement omponents C3a and C5a promote choroidal neovascularization. Proc Natl Acad Sci USA. 103(7):2328-2333. Epub 2006 Feb 1. PubMed Icon Google Scholar Icon