Research represents major breakthrough in macular degeneration

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.

add to favorites email to friend print save as pdf

Related Stories

Pivotal discoveries in age-related macular degeneration

Feb 06, 2011

A team of researchers, led by University of Kentucky ophthalmologist Dr. Jayakrishna Ambati, has discovered a molecular mechanism implicated in geographic atrophy, the major cause of untreatable blindness ...

First gene associated with dry macular degeneration found

Aug 27, 2008

In a study that underscores the important role that individual genetic profiles will play in the development of new therapies for disease, a multi-institutional research team – led by Kang Zhang, MD, PhD professor of ophthalmology ...

Vision loss slowed by encapsulated cell therapy

Apr 07, 2011

(PhysOrg.com) -- 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 ...

Recommended for you

Team explores STXBP5 gene and its role in blood clotting

21 minutes ago

Two independent groups of researchers led by Sidney (Wally) Whiteheart, PhD, of the University of Kentucky, and Charles Lowenstein, MD, of the University of Rochester, have published important studies exploring the role that ...

User comments