Age-Related Blindness: New Insights

Age-related macular degeneration (AMD) is a leading cause of blindness in the United States, and despite current treatments, its root causes and effective therapies remain elusive. A new study published in the journal Developmental Cell sheds light on the cellular processes involved in AMD, offering potential avenues for developing innovative treatments (1^✔ ✔Trusted Source
Human iPSC-based disease modeling studies identify a common mechanistic defect and potential therapies for AMD and related macular dystrophies

Go to source). "Current treatments for AMD have limited efficacy and often come with significant side effects," said Ruchira Singh, PhD, with the University of Rochester Flaum Eye Institute and Center for Visual Sciences, and lead author of the study. "Our research aims to identify novel therapeutic targets that could potentially halt the progression of this disease."

Stem Cells Unravel the Mystery of AMD

The study utilized human stem cells to model AMD, overcoming the limitations of previous research using animal models. By examining genes associated with both AMD and rarer inherited forms of blindness called macular dystrophies, the researchers identified a key protein involved in the early stages of the disease.

The retinal pigment epithelium (RPE), a layer of cells at the back of the eye, plays a crucial role in AMD. Over time, deposits of lipids and proteins, known as drusen, accumulate in the RPE. These deposits are often an early indicator of AMD.

By using a small molecule inhibitor to block the activity of the enzyme associated with inflammation, the researchers were able to reduce drusen formation in their model, suggesting that targeting this pathway could be a promising strategy for preventing AMD.

AMD affects the macula, the central part of the retina, leading to blurred or distorted vision. There are two main types: dry and wet AMD. Early detection and treatment can help manage symptoms and slow progression.

"Cellular pathways involved in drusen formation are key drivers of AMD progression," said Dr. Singh. "If we can halt the accumulation of drusen, we may be able to prevent the disease from progressing to a stage where vision loss occurs. This research offers hope for developing new treatments that could significantly improve the lives of millions of people affected by AMD."

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Additional co-authors include Sonal Dalvi, Michael Roll, Amit Chatterjee, Lal Krishan Kumar, Akshita Bhogavalli, Nathaniel Foley, Cesar Arduino, Whitney Spencer, and with the University of Rochester, Cheyenne Reuben-Thomas, Davide Ortolan and Kapil Bharti with the National Eye Institute, Alice Pebay with the University of Melbourne, and Bela Anand-Apte with the Cleveland Clinic. The research was supported by the National Eye Institute, ForeBatten Foundation, and Research to Prevent Blindness.

Reference:

1. Human iPSC-based disease modeling studies identify a common mechanistic defect and potential therapies for AMD and related macular dystrophies - (https://linkinghub.elsevier.com/retrieve/pii/S1534580724005331)

Source-Eurekalert