Blindness Fact Sheet

CIRM funds many projects seeking to better understand diseases of blindness and to translate those discoveries into new therapies.


Nearly a million Americans are blind, with another 2.4 million suffering significant visual impairment. While there are several causes of blindness, the leading cause of all visual impairment is age-related macular degeneration, which affects 1.7 million Americans. 

California’s stem cell agency funds research into potential therapies for three of the causes of blindness. All the research teams are seeking to use various forms of stem cells to rescue or replace cells in the eye damaged or threatened by the diseases. Several groups are working on ways to restore vision for people with age-related macular degeneration (AMD). Other projects are looking to preserve vision in patients with retinitis pigmentosa, and to restore clarity to the surface of eyes impacted by corneal disease.

Macular Degeneration

In AMD the layer of cells that support the photoreceptors is destroyed. Without this support system, the photoreceptors, the cells that actually allow us to sense light start to malfunction. CIRM-funded teams are looking at various methods of replacing this layer of support cells called RPE (retinal pigment epithelial) cells. Some are using embryonic stem cells as a starting point to generate new RPE cells. Others are using stem cells obtained by reprogramming adult cells to be like embryonic cells, which could potentially come from the patients’ themselves.

Retinitis Pigmentosa

Retinitis pigmentosa, an inherited and progressive vision loss that leaves most patients legally blind by mid-life, directly destroys the photoreceptors. CIRM-funded researchers are seeking to use stem cells to rescue the receptors from further damage and potentially replace them with new ones.

Limbal Stem Cell Deficiency

The cornea, the outer surface of the eye, is constantly refreshed by stem cells that reside in neighboring tissue. But some people just don’t have enough of these stem cells, called Limbal stem cells, to make enough new cornea cells. CIRM-funded researcher are trying to correct this condition, limbal stem cell deficiency, by retrieving the few existing limbal stem cells, and using various techniques to expand them in the laboratory until there are enough cells to rebuild a healthy cornea.

Some projects we fund are trying to take promising therapies out of the laboratory and closer to being tested in people. These Disease Team Awards encourage the creation of teams that have both the scientific knowledge and business skills needed to produce therapies that can get approval from the Food and Drug Administration (FDA) to be tested in people. In some cases, these awards also fund the early phase clinical trials to show that they are safe to use and, in some cases, show some signs of being effective.

Disease Team Awards

University of Southern California

This team is using embryonic stem cells to produce the support cells, or RPE cells, needed to replace those lost in AMD. Because these cells exist in a thin sheet in the back of the eye, they are assembling these sheets in the lab by growing the RPE cells on synthetic scaffolds. These sheets are then surgically implanted into the eye. They are testing the human embryonic stem cell-derived RPE cells in a Phase 1/2a clinical trial to treat the advanced dry form of AMD. 

University of California, Irvine

For retinitis pigmentosa, the team plans to use donor tissue to isolate cells that are part way down the path from neural stem cells to adult eye tissue. These retinal progenitor cells would be grown in large quantities in the lab and then injected into the eye. The team suggests the cells could help in two ways. They may be able to protect the photoreceptors not yet damaged by the disease, and they may be able to form new photoreceptors to replace those already lost. The team is testing the safety of transplanting human retinal progenitor cells into patients with RP in a phase 1/2 clinical trial.

Mark Humayun, who leads the USC disease team, discusses his approach to treating macular degeneration

CIRM Grants Targeting Vision Loss

Researcher name Institution Grant Title Approved funds
Mark Humayun University of Southern California Stem cell based treatment strategy for Age-related Macular Degeneration (AMD) $18,904,916
David Hinton University of Southern California Therapeutic potential of Retinal Pigment Epithelial cell lines derived from hES cells for retinal degeneration. $651,607
Martin Friedlander Scripps Research Institute Autologous Retinal Pigmented Epithelial Cells Derived from Induced Pluripotent Stem Cells for the Treatment of Atrophic Age Related Macular Degeneration $5,806,321
Gabriel Travis University of California, Los Angeles Development of a Stem Cell-based Transplantation Strategy for Treating Age-related Macular Degeneration $5,487,136
Peter Coffey University of California, Santa Barbara Development of Cellular Therapies for Retinal Disease $4,850,116
Henry Klassen University of California, Irvine Retinal progenitor cells for treatment of retinitis pigmentosa $17,144,825
Deepak Lamba Buck Institute for Age Research 3D Modeling of Retina using Polymer Scaffolds for Understanding Disease Pathogenesis $1,212,553
Kang Zhang University of California, San Diego Generation of fibroblast cell lines in patients with common blinding eye diseases $1,034,425
Thomas Novak Cellular Dynamics International Generation and characterization of high-quality, footprint-free human induced pluripotent stem cell lines from 3,000 donors to investigate multigenic diseases $16,000,000
Cristian Perez Coriell Institute for Medical Research The CIRM Human Pluripotent Stem Cell Biorepository – A Resource for Safe Storage and Distribution of High Quality iPSCs $9,942,175
Magdalene Seiler University of California, Irvine Restoring vision by sheet transplants of retinal progenitors and retinal pigment epithelium (RPE) derived from human embryonic stem cells (hESCs) $3,998,948
Mark Humayun University of Southern California Phase 1 Safety Assessment of CPCB-RPE1, hESC-derived RPE Cell Coated Parylene Membrane Implants, in Patients with Advanced Dry Age Related Macular Degeneration $17,128,661
Sophie Deng University of California, Los Angeles Regeneration of Functional Human Corneal Epithelial Progenitor Cells $697,507
David Schaffer University of California, Berkeley Engineered Biomaterials for Scalable Manufacturing and High Viability Implantation of hPSC-Derived Cells to Treat Neurodegenerative Disease $1,239,276
Sophie Deng University of California, Los Angeles Regeneration of Functional Human Corneal Epithelial Progenitor Cells $1,524,947
Shaomei Wang Cedars-Sinai Medical Center IND-enabling study of subretinal delivery of human neural progenitor cells for the treatment of retinitis pigmentosa $4,954,514
Henry Klassen University of California, Irvine Human retinal progenitor cells as candidate therapy for retinitis pigmentosa $1,803,768

CIRM Macular Degeneration Videos

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