Research represents major breakthrough in macular degeneration

April 26, 2012

University of Kentucky researchers, led by Dr. Jayakrishna Ambati, have made a major breakthrough in the "dry" form of age-related macular degeneration known as geographic atrophy (GA). GA is an untreatable condition that causes blindness in millions of individuals due to death of retinal pigmented epithelial cells. The paper, "DICER1 loss and Alu RNA Induce Age-Related Macular Degeneration via the NLRP3 Inflammasome and MyD88," was published in the April 26 online edition of the premier journal Cell.

Ambati, professor of physiology, and professor and vice chair of ophthalmology and visual sciences at UK, is a leader in the field of macular degeneration research. Previous research from the Ambati laboratory published in the journal Nature showed that in human eyes with geographic atrophy there is a deficiency of the enzyme DICER1, leading to accumulation of toxic Alu in the retinal pigmented epithelium. The Cell paper shows that when these RNAs build up in the eye they trigger activation of an immune complex known as the NLRP3 inflammasome. In turn, this leads to the production of a molecule known as IL-18, which causes death of retinal pigmented and vision loss by activating a known as MyD88.

Importantly, Ambati and colleagues found evidence that activity of the inflammasome, IL-18, and MyD88 were all increased in human eyes with GA. They then showed that blocking any of these components could prevent retinal degeneration in multiple . The researchers are excited that blocking these pathways could herald a new potential therapy for GA, for which there is no approved treatment.

Explore further: Vision loss slowed by encapsulated cell therapy

Related Stories

Vision loss slowed by encapsulated cell therapy

April 7, 2011

( -- A phase 2 clinical trial for the treatment of a severe form of age-related macular degeneration called geographic atrophy (GA) has become the first study to show the benefit of a therapy to slow the progression ...

Recommended for you

An accessible approach to making a mini-brain

October 1, 2015

If you need a working miniature brain—say for drug testing, to test neural tissue transplants, or to experiment with how stem cells work—a new paper describes how to build one with what the Brown University authors say ...

Tension helps heart cells develop normally in the lab

October 1, 2015

The heart is never quite at rest, and it turns out that even in a lab heart cells need a little of that tension. Without something to pull against, heart cells grown from stem cells in a lab dish fail to develop normally.

Dormant viral genes may awaken to cause ALS

September 30, 2015

Scientists at the National Institutes of Health discovered that reactivation of ancient viral genes embedded in the human genome may cause the destruction of neurons in some forms of amyotrophic lateral sclerosis (ALS). The ...


Please sign in to add a comment. Registration is free, and takes less than a minute. Read more

Click here to reset your password.
Sign in to get notified via email when new comments are made.