• The National Eye Institute

    The National Eye Institute (NEI) has selected ten winning submissions from a pool of nearly 500 entries to its Audacious Goals challenge, a nationwide competition for compelling, one-page ideas to advance vision science. 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., outside Washington, D.C.

    Winning ideas include restoring light sensitivity to the blind, precision correction of defective genes, and growing healthy tissue from stem cells for ocular tissue transplants.

    "The Audacious Goals initiative was born out of the NEI strategic planning process, however it is much more than a standard strategic planning exercise," said Paul A. Sieving, M.D., Ph.D., NEI director. "We are envisioning the future. When we look back 10 to 12 years from now, what do we want to have accomplished? The Audacious Goals initiative will help propel us into that future."


    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 winning ideas.


    The winners and their audacious ideas are:


    Dennis Clegg, Ph.D.

    University of California, Santa Barbara, Calif.

    Regenerative Therapy for Retinal Disease

    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.

    Robert Duvoisin, Ph.D.

    Oregon Health and Science University,
    Portland, Ore.


    Restoration of Vision by Opto-electronic Stimulation

    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.

    Yingbin Fu, Ph.D.

    University of Utah, Salt Lake City, Utah

    Precise Gene Editing In Vivo

    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.


    Steven Pittler, Ph.D.

    University of Alabama, Birmingham, Ala.

    Using Molecular Scissors Genome Editing to Cure Ocular Genetic Disease

    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.


    Rajesh Rao, M.D.

    Washington University School of Medicine in
    St. Louis and The Retina Institute, St. Louis, Mo.


    An Audacious Goal: Reprogramming the Retina

    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.

    Tonia Rex, Ph.D.

    Vanderbilt University, Nashville, Tenn.

    Functional and Structural Neuroregeneration

    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.

    Julia Richards, Ph.D.

    University of Michigan, Ann Arbor, Mich.

    Fountains of Youth for the Eye

    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.

    Jeffrey Stern, M.D., Ph.D.

    Capital Region Retina, PLLC, Albany, N.Y.

    Endogenous Retinal Repair: Releasing our Inner Salamander

    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.

    Russell Van Gelder, M.D., Ph.D.

    University of Washington, Seattle, Wash.

    Reversing Retinal Blindness Using Small Molecules

    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.

    Janey Wiggs, M.D., Ph.D.

    Massachusetts Eye and Ear Infirmary, Harvard Medical School, Boston, Mass.

    Vision BioBank - A Network of Ocular Phenotyping Centers Using Genomic and Epidemiologic Data to Promote Personalized Ophthalmology

    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.


    Next Steps

    The winners have been invited to present their ideas at the NEI Audacious Goals Development Meeting, which will include about 200 vision researchers, patient advocates, ophthalmologists, and optometrists from the U.S. and abroad. The selected ideas will be discussed intensively for further expansion, development, and refinement. Following the meeting, NEI staff and members of the National Advisory Eye Council will finalize and publish a set of the most compelling audacious goals for the institute and the broader vision research community to pursue over the next decade.

    For more information, visit the Audacious Goals website.