Sunday, February 10, 2013

NEI Rewards the Audacious

The National Eye Institute (NEI) has selected 10 submissions from some 500 entries for its Audacious Goals challenge. The NEI Challenge is part of a government-wide effort to bring the best ideas to bear on the nation's most pressing challenges. During the judging process, more than 80 experts in the vision community helped narrow the field to 81 final candidates. A federal panel consisting of 13 clinicians and scientists then selected the following winning ideas:
  • Regenerative Therapy for Retinal Disease (Dennis Clegg, Ph.D., University of California, Santa Barbara): To treat degenerative retinal disease with an off-the-shelf tissue graft that could be implanted in the back of the eye to replace cells lost to disease.
  • Restoration of Vision by Opto-electronic Stimulation (Robert Duvoisin, Ph.D., Oregon Health and Science University, Portland): To restore vision by making nerve cells in the eye sensitive to light so that images captured by a camera can be converted to nerve signals that are sent to the brain.
  • Precise Gene Editing In Vivo (Yingbin Fu, Ph.D., University of Utah, Salt Lake City): To permanently correct any disease-associated mutations in a patient through the use of molecules that are specially designed to target mutated DNA sequences and that can be delivered safely and efficiently into the eye.
  • Using Molecular Scissors Genome Editing to Cure Ocular Genetic Disease (Steven Pittler, Ph.D., University of Alabama, Birmingham, AL): To permanently correct gene defects in patients at the site of the mutation using molecules that act like scissors to precisely replace genome errors with the correct DNA sequence.
  • An Audacious Goal: Reprogramming the Retina (Rajesh Rao, M.D., Washington University School of Medicine in St. Louis and The Retina Institute, St. Louis): To directly reprogram easy-to-isolate skin or blood cells to retinal cells using gene therapy and other techniques to enable repair strategies for degenerative retinal diseases.
  • Functional and Structural Neuroregeneration (Tonia Rex, Ph.D., Vanderbilt University, Nashville): To restore functional vision in patients who experience loss of axons-the threadlike extensions of a nerve cell that conduct electrical impulses-from the optic nerve as a result of traumatic optic neuropathy or glaucoma by complete axon regeneration.
  • Fountains of Youth for the Eye (Julia Richards, Ph.D., University of Michigan, Ann Arbor): To turn back the aging process in the eye so that ocular diseases like age-related macular degeneration or glaucoma start 10, 20, or 30 years later than they now do.
  • Endogenous Retinal Repair: Releasing our Inner Salamander (Jeffrey Stern, M.D., Ph.D., Capital Region Retina, PLLC, Albany, NY): To repair the retina by activating stem cells residing within the eye, awakening reparative processes that occur naturally in amphibians and other animals but which lay dormant in human patients.
  • Reversing Retinal Blindness Using Small Molecules (Russell Van Gelder, M.D., Ph.D., University of Washington, Seattle): To restore vision to patients with retinal diseases through the use of a photoswitch, a small molecule that is chemically modified to become active or inactive after exposure to certain wavelengths of light.
  • Vision BioBank - A Network of Ocular Phenotyping Centers Using Genomic and Epidemiologic Data to Promote Personalized Ophthalmology (Janey Wiggs, M.D., Ph.D., Massachusetts Eye and Ear Infirmary, Harvard Medical School, Boston) : To create a network of biobanks that collect corresponding phenotype (physical characteristics) and genotype (genetic) data of people with certain eye diseases; the biobanks could be used for a wide range of studies, including the development of sensitive and specific gene tests that could accurately determine a person's risk for glaucoma, age-related macular degeneration, diabetic retinopathy, and other common complex blinding diseases as well as their likely response to certain therapies.

Each winner will receive a $3,000 prize plus travel expenses to attend the NEI Audacious Goals Development Meeting, Feb. 24-26, 2013, at the Bolger Conference Center in Potomac, MD. There, some 200 vision researchers, patient advocates, ophthalmologists, and optometrists from the U.S. and abroad will discuss some of the ideas. Then NEI staff and members of the National Advisory Eye Council will publish the most compelling audacious goals for the institute and the broader vision research community. For more information, visit

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