Therapeutic MyD88 Inhibition in a Novel Model of Dry Age-Related Macular Degeneration

  • Ambati, Jayakrishna (PI)

Grants and Contracts Details


We are applying for this Carl Marshall Reeves & Mildred Almen Reeves Foundation grant in order to fill the therapeutic void of “dry” age-related macular degeneration (AMD); there are no approved therapies for this disease, which is the leading cause of irreversible blindness in the United States. Our laboratory has been at the forefront of research discoveries in AMD, and we believe that our recent work has set the stage for a major breakthrough in finally delivering the first effective treatment for patients with dry AMD. Our research has revealed a key mechanism of AMD pathogenesis, and by blocking a key signaling molecule (MyD88) in this pathway in a mouse model of AMD, we were able to successfully prevent the characteristic RPE cell death that defines dry AMD. Still, effective translation of this therapeutic strategy to humans will require additional efficacy safety studies in non-human primates. Furthermore, the use of NHPs is essential to ascertaining the therapeutic potential of MyD88 inhibition because the Alu RNA sequences that cause RPE cell death via MyD88 activation are unique to primates. Therefore, the main purpose of the current proposal is to design novel therapeutics to inhibit MyD88 activity in DICER1/Alu dysregulation-mediated RPE death in the non-human primate model of dry AMD.
Effective start/end date12/1/128/31/13


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