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NEI Audacious Goal and Research Priorities
Regenerate Neurons and Neural Connections in the Eye and Visual System
- 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
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
- 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 Updated: May 2013
