Can Eyesight be restored with genetically engineered stem cells?
Macular degeneration symptoms can develop at an early age, and while it doesn’t cause pain it leads to visual distortion, such as straight lines seeming bent.
Reduced central vision in one or both eyes. The need for brighter light when reading or doing chores, and increased difficulty in adapting to low light levels when entering dimly light areas. It also includes increased blurriness when reading printed words, as well as difficulty recognizing the faces of people on the streets.
Over time, without proper care and attention, this can lead to further retinal degeneration that in extreme cases leads to blindness. Researchers are keen on finding solutions and are now looking into gene and stem cell therapies for a cure.
The Centre for Genomic Regulation in Barcelona, Spain developed a stem cell treatment for retinal degeneration that focuses on chemokine receptors, Ccr5 and Cxcr6, which they identified to be important players in cell signaling. Engineering mesenchymal stem cells from bone marrow to an excessive amount of Ccr5 and Cxcr6 and transplanted them into models of retinal degeneration. That preserved the function of degenerating retinal tissue. The study was published in the journal of Molecular Therapy.
Scientists studied stem cells extensively for the last 20 years, and know that it contains properties that help retinal cells repair themselves. They release molecules that naturally protect the neural cells and reduce inflammation. However, there are challenges when it comes to using stem cells to treat eye disorders. Cells struggle to migrate to the eyes and integrate themselves into the retina.
“After the cells are transplanted they need to reach the retina and integrate through its layers,” Cosma said. “Here we have found a way to enhance this process using stem cells commonly found in the bone marrow, but in principle can be used with any transplanted cells.”-CRG research professor Pia Cosma
Mesenchymal stem cells offer advantages when it comes to solving eye diseases. It has the ability to transform itself into retinal cells and grow outside the body. When these evolved cells are transplanted into humans and mouse test subjects, the cells migrate in high numbers to deteriorating retinal tissue and prevent the demise of retinal cells.
Another team, called Mount Sinai is working on a therapy that stimulates and genetically reprograms dormant Müller glial cells in the retina to repair the damage. The University of Southern California researchers were also able to create retinal implants from embryonic stem cells. It was proven during a clinical trial that it could improve the eyesight of people with advanced dry AMD.
The next step for the CRG team is to investigate whether alternative cell-modification methods might improve their Ccr5- and Cxcr6-targeted therapy. They modified the stem cells for their study with lentiviruses, which have been used in experimental gene therapies.
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