Scientists develop eye drops that slow vision loss in animals with inherited retinal diseases
Researchers have developed eye drops that slow vision loss in animal models of retinitis pigmentosa. Containing a peptide from PEDF, the drops reach the retina quickly, showing potential for human trials without toxicity or side effects.
FollowResearchers have developed a groundbreaking eye drop treatment that slows vision loss in animal models of retinitis pigmentosa, a group of inherited diseases that cause progressive blindness in humans. The new findings, published in Communications Medicine, offer hope for patients with degenerative retinal disorders.
The eye drops contain a small protein fragment derived from pigment epithelium-derived factor (PEDF), a natural protein found in the eye that helps protect retinal cells. While not a cure, the treatment has shown promise in slowing the progression of multiple degenerative retinal diseases, including retinitis pigmentosa and dry age-related macular degeneration (AMD).
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"Given these results, we're excited to begin trials of these eye drops in people," said Patricia Becerra, Ph.D., senior author of the study and chief of the Section on Protein Structure and Function at the National Eye Institute (NEI), part of the NIH.
Degenerative retinal diseases, including retinitis pigmentosa and AMD, are driven by cellular stress, which leads to the gradual loss of photoreceptor cells, ultimately resulting in vision impairment and blindness. Despite the varying genetic causes of these diseases, high levels of cellular stress are a common factor.
In this study, led by first author Alexandra Bernardo-Colon, researchers developed two eye drop formulations containing short peptides designed to protect the retina. One peptide, called "17-mer," consists of 17 amino acids from the active region of PEDF. The second, H105A, is a modified version that binds more strongly to the PEDF receptor, potentially enhancing its protective effects.
Tests on mice showed that after application, the peptides reached high concentrations in the retina within 60 minutes and remained active for up to 48 hours. Importantly, the treatment did not cause any toxicity or adverse side effects.
With these promising results, researchers are now preparing for human clinical trials, aiming to offer a non-invasive and accessible treatment to slow vision loss in people with retinal degeneration.
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