Hereditary retinal diseases (RIDs) are a group of genetically and clinically homogeneous diseases characterized by progressive retinal damage resulting in loss of vision. The worldwide incidence of IRD is approximately 1 in 2000 people. These disorders are among the leading causes of blindness worldwide.
While the introduction of gene therapy was an important development, its effectiveness has been blunted by the sheer extent of genetic heterogeneity, with over 260 genes involved in IRD. This limits the widespread application of gene therapy to all IRDs. In addition, gene therapy has limited efficacy in clinical cases of advanced retinal degeneration in which significant cell death of photoreceptors has already occurred. Photoreceptor cells are found in the retina and respond to light, converting it into electrical signals that activate physiological chain reactions. These signals are sent by the optic nerve to the brain for processing.
With the advent of induced pluripotent stem cell (iPSC) and embryonic stem cell (ESC) technology, regenerative stem cell therapy has the potential to be an alternative treatment for end-stage retinal degeneration, regardless of the defect. underlying genetics. Regenerative retinal therapies are therefore very promising for the treatment of IRD. Studies in animal models of IRD have suggested visual improvement after transplantation of retinal photoreceptor precursors, although there is limited evidence on the ability of these transplants to repair retinal damage in higher mammals.
A study recently published in the journal Stem cell research and therapy headed by Assistant Professor Su Xinyi of the Ophthalmology Department of NUS Yong Loo Lin Faculty of Medicine, examines the therapeutic potential of photoreceptor precursors derived from clinically compliant iPSCs. The study demonstrated the safety and therapeutic potential of clinically compliant iPSC-derived photoreceptor precursors as a cell replacement source for future clinical trials. These include the completion of a first human clinical trial for photoreceptor precursor transplantation in Singapore, in collaboration with RxCELL, a biotechnology company focused on therapeutic applications of iPSCs.
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