Stem cells can restore vision in inherited retinal diseases

A new study of photoreceptor precursors derived from induced pluripotent stem cells (iPSCs) has demonstrated their safety and therapeutic potential as a source of replacement for damaged cells for future clinical trials.

Inherited retinal diseases (IRDs) are a group of genetic diseases characterized by progressive damage to the retina that leads to loss of vision. IRDs affect approximately 1 in 2,000 people worldwide.

Even though gene therapy today demonstrates significant advances in medicine, it is ineffective in treating inherited retinal diseases. This is due to the high degree of genetic heterogeneity of IRDs, which can be caused by mutations in more than 260 genes.

This fact limits the widespread use of gene therapy for all cases of IRDs. In addition, gene therapy has limited efficacy in clinical cases of progressive retinal degeneration in which a significant number of photoreceptors have already died. Photoreceptor cells are located in the retina and respond to light by converting it into electrical signals that can stimulate biological processes.

With the advent of technologies for creating induced pluripotent stem cells (iPSCs) and embryonic stem cells (ESCs), regenerative stem cell therapy may become an alternative treatment for end-stage retinal degeneration, regardless of the genetic mutations that cause it.

Thus, retinal regenerative therapy opens excellent prospects for the treatment of IRDs. Studies on crab-eating macaque models with inherited retinal diseases have shown improved vision after retinal photoreceptor precursors transplantation. Until now, there has been little evidence that grafts can repair retinal damage only in apes.

A new study, recently published in the journal Stem Cell Research and Therapy, examined the therapeutic potential of photoreceptor precursors derived from clinically compliant iPSCs.

A study led by Assistant Professor Su Xinyi from the Department of Ophthalmology at the Yong Loo Lin School of Medicine at the National University of Singapore demonstrated iPSC-derived photoreceptor precursors’ safety and therapeutic potential as a source of cell replacement for future clinical trials. These include conducting the first clinical trials of photoreceptor precursor transplantation in Singapore in collaboration with RxCELL, a biotechnology company specializing in the therapeutic use of induced pluripotent stem cells.