The Development of a Transplant-Independent Therapy for RPE Dysfunction

The Development of a Transplant-Independent Therapy for RPE Dysfunction is sponsored by BrightFocus Foundation. This grant supports research focusing on transplant-independent therapies that restore dysfunctional RPE cells and prevent AMD progression. The study is unique in rejuvenating dysfunctional RPE cells themselves in vivo using epigenetic reprogramming.

The Development of a Transplant-Independent Therapy for RPE Dysfunction Grants > The Development of a Transplant-Independent Therapy for RPE Dysfunction Macular Degeneration Research Grant The Development of a Transplant-Independent Therapy for RPE Dysfunction Innovative Approaches to Macular Degeneration Treatments Shintaro Shirahama, MD, PhD Schepens Eye Research Institute of Massachusetts Eye and Ear Macular Degeneration Research July 01, 2024 - June 30, 2026 This grant is made possible by the support of the Free Family Foundation.

Bruce Ksander, PhD, Schepens Eye Research Institute of Massachusetts Eye and Ear The research team hypothesizes that epigenetic reprogramming of dysfunctional retinal pigment epithelium cells will reverse the biological age of the cells, ultimately improving visual function.

Aging is a critical risk factor for developing age-related macular degeneration (AMD), which implies that young retinal pigment epithelium (RPE) cells can successfully combat the underlying causes of AMD. Conversely, as RPE cells age, they undergo alterations, rendering them more susceptible to AMD pathogenesis.

Dr. Shirahama’s team found that epigenetic reprogramming through OSK (Oct4, Sox2, and Klf4) transduction could reverse the cellular biological age and restore function. Therefore, if they can reverse the biological age of RPE cells, these younger cells could successfully combat the disease, stopping further progression and restoring functional vision.

Most other studies focus on differentiating stem cells to form RPE monolayers to replace dysfunctional RPE. This study is unique in rejuvenating dysfunctional RPE cells themselves in vivo using epigenetic reprogramming through OSK introduction. Loss of retinal pigment epithelium (RPE) function is a fundamental cause of dry and wet age-related macular degeneration (AMD).

A still treatment is the transplantation of RPE sheets derived from induced pluripotent stem cells (iPSCs). However, significant problems exist in regenerating the RPE layer using this transplantation method. This study will bring transplant-independent therapies that restore dysfunctional RPE cells and prevent AMD progression.

Macular Degeneration Research New Drug Delivery Approach to Transform Macular Degeneration Treatment July 01, 2025 - June 30, 2028 University of New South Wales Macular Degeneration Research A Novel Implantable Device to Treat Wet Macular Degeneration July 01, 2025 - June 30, 2028 Macular Degeneration Research Innovative Night Vision Tests for Age-Related Macular Degeneration July 01, 2024 - June 30, 2027 Institute of Molecular and Clinical Ophthalmology Basel (Switzerland) News Featuring This Grantee Turning Back the Biological Clock on Aged and Injured Eye Cells A de-aging technique offers hope for halting damage in early macular degeneration.