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Credit: https://www.fiercebiotech.com/research/restoring-eyesight-genetically-engineered-stem-cells

Thousands May Have Sight Restored Thanks to Stem Cells

December 2, 2020

  • New gene therapy and stem cell research innovations have led to new breakthroughs in treating eye diseases. 
  • Scientists have created stem cells that can be transplanted into the eyes of patients with age-related macular degeneration and retinitis pigmentosa to improve vision. 
  • Researchers have also reprogrammed native glial cells in a patient’s eye to initiate the repair of the retina. 
Millions worldwide experience progressive vision loss because of either age-related macular degeneration (AMD) or retinitis pigmentosa (RP). These similar but distinct diseases lead to atrophy of the eye’s retina, or light-sensitive area. Slowing or reversing retinal atrophy has been a treatment goal for decades, but only recently have innovations in gene and stem cell technology allowed for new, viable treatment paths.  Scientists at the Centre for Genomic Regulation in Barcelona targeted two essential cell signaling receptors in their research, Ccr5 and Cxcr6. The researchers developed mesenchymal stem cells with an abundance of the two receptors, more than would be found in a natural cell. The lab-grown cells with extra receptors proved more resistant to the atrophy typical of AMD and RP.  While the scientists successfully produced a resilient cell, challenges remain when determining how to introduce the stem cells into the eye of a patient with retinal atrophy. Further modification and engineering of the lab-grown cells will lead to stem cells that are highly specific to retinal tissue and migrate quickly to the damaged areas of the retina.  Another team at the National Institute of Health’s National Eye Institute used human skin cells to create retinal cells eligible for transplant. The skin cells are first transformed into pluripotent stem cells and then guided to develop into rod photoreceptors, light-sensitive cells found in the retina. These lab-grown cells are designed to be transplanted into the retina of an AMD or RP patient.  Yet another technique aims to use the eye’s own cells to repair damage. Researchers at Mount Sinai have developed a therapy that reprograms dormant glial cells in the retina to improve atrophic areas of the retina. Embryonic stem cells also show promise, with researchers at the University of Southern California demonstrating in a small trial of AMD patients that stem cell therapy could improve vision. Through stem cells and genetic reprogramming, scientists have introduced several new routes to restoring the vision of thousands.  Credit: https://www.fiercebiotech.com/research/restoring-eyesight-genetically-engineered-stem-cells 

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