NEI Audacious Goal and Research Priorities

Regenerate Neurons and Neural Connections in the Eye and Visual System

Target tissues: Photoreceptors and Retinal Ganglion Cells (see

an eye

  • Establishing functional neural connections would be a pinnacle achievement for regenerative medicine in the eye.
  • Paradigm shift to create a new understanding of plasticity and regeneration.
  • Addresses the pathogenesis of many ocular and vision diseases.
  • Provide a model for regenerative therapies beyond vision, for treating CNS disease and spinal cord injury.

Examples of what we will need to know:

  • What trophic factors will stimulate and guide axons to specific targets in the brain?
  • How do synapses form in an adult?
  • What steps are required for exogenous repair?
  • Can we activate latent endogenous cells to replace lost host neurons?
  • How do we control immune responses and ensure safety and efficacy?
  • How do we monitor in vivo for functional success?

Molecular Therapy for Eye Disease

New Funding Opportunity:
PA-13-283 NEI Audacious Goals Initiative in Vision Research High Priority Research Area: Molecular Therapies for Eye Disease (R01)

Why Now?

  • Genetic and cellular bases of many eye diseases are rapidly becoming understood.
  • Proof of concept demonstrated for ocular gene therapy.
  • New and promising technologies are coming on line for precise gene correction in vivo.
  • Molecular design and biology of light-sensitive molecules is feasible.

Examples of what we will need to know:

  • Define and prioritize among disease targets.
  • Explore in vivo gene editing and correction tools for the eye.
  • Define unique markers on a large scale for specific ocular cell types.
  • Provide targeted reagents for specific cell types, and demonstrate regulatable, high-efficiency therapy.

Intersection of Aging & Biological Mechanisms of Eye Disease

New Funding Opportunity:

PA-13-332 Audacious Goals Initiative High Priority Research Area: Intersection of Aging and Biological Mechanisms of Eye Disease (R01)

  • Understand how the biology of aging contributes to disease and the course of disease.
  • Evaluate how the failure of homeostatic processes causes or allows the transition from aging to early disease.
  • Define biological staging of disease to understand pathophysiology toward prevention and therapy.

Why Now?

  • Genetic and epigenetic risk factors are already identified for ocular diseases of aging.
  • Longevity field has provided proof of concept that murine life span can be extended.
  • New technologies allow earlier detection of disease.

Examples of what we will need to know:

  • Normal homeostasis of ocular tissues and deviations that contribute to disease.
  • Biological predictors of early onset disease.
  • Targets for early intervention.
  • Biological pathways and “zones of transition” from normal aging to disease?
Last Reviewed: 
February 2014