Gene-independent Therapeutic Interventions in Degenerating Photoreceptors – Physician’s Weekly

One of the leading causes of blindness in developed nations is neurodegenerative diseases of the retina. Therapeutic treatments are often unavailable, despite their critical role in enhancing patients lives. Significant interest existed in developing gene therapies for a group of diseases for which the underlying genes had already been identified; these diseases primarily affected photoreceptors. But gene-indepdendent approaches were required depending on the type and severity of the disease. Researchers are exploring several potential methods to slow the spread of disease or preserve retinal function. Neuroprotection, direct reprogramming of damaged photoreceptors, non-coding (ribonucleic acid) RNAs, optogenetic strategies for creating artificial photoreceptors, and cell replacement therapies are examples of interventions. Recent advances have been made, such as the first optogenetic application to a blind patient whose visual function was partially recovered by targeting retinal ganglion cells. RPE (Retinal Pigment Epithelium) cell transplantation therapies are also being studied in clinics, and they hold great promise for restoring sight to the visually impaired. Human embryonic stem cells were used to create these cells. There has been extensive testing of photoreceptor replacement therapies in pre-clinical models. Taking advantage of advances in genetic engineering, optogenetics, and stem-cell research, this is just the beginning of promising new cures. Here is a summary of the current state of gene-independent therapeutics and a highlight of the recent advances that have been made. Since photoreceptors are essential for light perception but are particularly susceptible to degenerative diseases, they are the primary focus of this research.

Source: sciencedirect.com/science/article/pii/S1350946222000258

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Gene-independent Therapeutic Interventions in Degenerating Photoreceptors - Physician's Weekly