LA JOLLA, CA October 23, 2013 A promising technique for treating human eye disease has proven effective in preclinical studies and may lead to new treatments to prevent blindness, according to experiments conducted at The Scripps Research Institute (TSRI) in La Jolla, California.
The studies involved controlling the actions of microRNAs, tiny pieces of RNA that were once considered to be "junk" but are now known to fine-tune gene activation and expression. The researchers showed that treating mice with short RNA strands that precisely target and inhibit microRNAs ("antimicroRNAs") can stop the aberrant growth of blood vessels ("neovascularization").
It is this abnormal proliferation of vessels that exacerbates vision loss in neovascular eye diseases like "wet" macular degeneration and diabetic retinopathy, two of the leading causes of blindness.
Described in the cover story of the November issue of the Journal of Clinical Investigation, the microRNA treatments blocked aberrant vessel growth without damaging existing vasculature or neurons in three separate models of neovascular eye diseasea proof-of-principle that suggests future treatment based on the same approach may be effective in humans.
"We believe that targeting and inhibiting the action of microRNAs involved could represent a novel and effective way to treat a broad range of neovascular eye diseases such as diabetic retinopathy, macular degeneration and macular telangiectasia," said TSRI Professor Martin Friedlander, MD, PhD, who was senior author of the study. "We are excited about this approach to halting abnormal blood vessel growth without inducing off-target side effects."
The work is the first published result of a five-year, $10.2 million grant awarded last year by the National Eye Institute of the National Institutes of Health. The grant aims to harness the potential of microRNAs to stop abnormal blood vessel sprouting in the back of the
|Contact: Mika Ono|
Scripps Research Institute