Rebuilding the blood–retina barrier to learn how APOE gene types affect macular degeneration

Reconstruction of a human blood-retina barrier and perfused vascular system to investigate the role of APOE variants in age-related macular degeneration

NIH-funded research Icahn School of Medicine at Mount Sinai · NIH-11270833

Researchers will grow a human retinal tissue with blood-vessel channels to learn how different APOE gene types influence age-related macular degeneration in people.

Quick facts

Grant typeR21 grant
Study typeNIH-funded research
Funding institutionIcahn School of Medicine at Mount Sinai NIH-funded
Lab location1 site (New York, United States)
Project IDNIH-11270833 on NIH RePORTER

What this research studies

This project will build a lab-grown human outer blood–retina barrier using human induced pluripotent stem cells that includes the retinal pigment epithelium and a choriocapillaris-like, perfused vascular compartment. The team will create versions of the tissue carrying different APOE gene variants and trigger lipid-rich sub‑RPE deposits similar to drusen to see how those variants change lipid handling and damage. They will optimize the system so it can be assembled reliably from cryopreserved cells and used for controlled comparisons. The goal is a human-relevant testbed to study mechanisms and to screen potential treatments that target lipid pathways in AMD.

Who could benefit from this research

Good fit: Ideal participants would be people with age-related macular degeneration or individuals known to carry APOE risk variants who are willing to donate blood or skin samples for iPSC generation.

Not a fit: People seeking an immediate treatment or vision improvement should not expect direct benefit from this laboratory-based research.

Why it matters

Potential benefit: If successful, this could clarify how APOE variants drive drusen formation and provide a human model to speed development of targeted therapies for AMD.

How similar studies have performed: Related iPSC-derived retinal models have reproduced sub‑RPE deposits and are promising, but directly comparing APOE variants in a perfused blood–retina barrier is a relatively new approach.

Where this research is happening

New York, United States

Researchers

About this research

  1. This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
  2. Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
  3. For full project details, budget, and progress reports, visit the official NIH RePORTER page below.
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.