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Gene Research Major Breakthrough in Diabetic, Elderly Eye Disease

Posted by Jane Akre
Tuesday, March 18, 2008 11:41 AM EST
Category: Major Medical
Tags: Diabetes, FDA and Prescription Drugs, Macular Degeneration, Gene Therapy

Gene therapy a major breakthrough for macular degeneration.


  • University of Utah summary of study here
  • Injuryboard on diabetes here

IMAGE SOURCE:© iStockphoto/ Sugar pond

Age-related macular degeneration (AMD) is a very common affliction and accounts for most cases of blindness over the age of 50.

As one ages, the center of the eye, the macula area of the retina, thins out. That can mean an inability to read or see far distances.

Now gene research is holding some promise that could help AMD sufferers and those afflicted with diabetic retinopathy, which is a complication of diabetes that can lead to blindness.

The gene is called Robo4, which plays an important role in stabilizing blood vessels telling them not to grow.

University of Utah researchers activated the Robo4 gene in mice that were bred to have the effects of macular degeneration, to make the eye vessels stop leaking and to stabilize. Leaking blood vessels are thought to contribute to AMD.

Robo4, a protein, actually countered another function in the eye, a chemical called vascular endothelial growth factor (VEGF) which, when signalling the creation of new blood vessels also is causing leaking eye vessels.

Robo4 stopped the growth of the vessels and then reversed damage something seen as a "major breakthrough" in reversing blindness if it works in humans.

Clinical trials could take place within five years.

“This is a major breakthrough in an area where the advances have been minimal. We are excited about taking this opening and moving the frontier forward with real hope for patients who have but few, often disappointing, options,” Dr. Olson who heads the university department overseeing research.

“Everything in biology has a yin (negative) and a yang (positive), and in the previous paper on netrins we brought attention to a new signaling pathway that induces vessels and nerves to grow,” Dr. Dean Li, lead researcher, says in a statement.

“Robo4 is the yin to that process, preventing new vessel growth by stabilizing the integrity of mature blood vessels.”

The study was published in the March 16 online edition of Nature Medicine.

This is good news for the approximately 10 million people who suffer blindness from AMD or diabetes. See the Macular Degeneration Overview for more information. #

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