RECENT THERAPEUTIC ADVANCES & PRECLINICAL PHASES OF RETINITIS PIGMENTOSA

Abstract

Retinitis pigmentosa (RP) is a group of hereditary illnesses characterized by a slow loss of retinal photoreceptors, which impairs a long-term vision. It is among the most prevalent forms of hereditary retinal dystrophy and has a sizable cost impact on both the individuals affected and the community as a whole. The typical symptoms of this disorder include nyctalopia, loss of the concentric visual field, and finally loss of the bilateral central vision.The primary effect is a progressive loss of vision. One of the main causes of vision loss and blindness in persons under 60, it affects roughly 1.5 million people globally. There is presently no known cure for RP, and the only approved gene therapy, voretigeneneparvovec, is only given to a tiny subset of patients with known RPE65 mutations. Retinoids, vitamin A supplements, sun protection, visual aids, and medical and surgical treatments to treat ocular comorbidities are now the only available therapies, however these merely serve to halt the diseases progression. Given the limited therapeutic landscape, it is vital to develop fresh, personalized therapy modalities that focus on degeneration of the retina. Although the variety of the gene mutations involved makes it difficult to identify a target treatment for RP, new fundamental research indicates an improvement towards comprehending the causes of retinal degeneration. Discovering novel molecular treatments that can specifically target specific receptors or signaling pathways lays the foundation for more effective medication development. The most recent advancements in potential RP treatments are highlighted in this article, with an emphasis on preclinical stage foundational research on molecular targets, which will form the basis for subsequent drug development. We will discuss the alterations that take place to the molecular pathways connected to the growth of RP, with a focus on the ER stress and apoptotic pathways, redox balance maintenance, and genomic stability. Subsequently proceed over the treatment modalities being looked at, like gene and cell therapy, as well as the most recent studies that are discovering new potential drug targets for RP.