Restoring eyesight with genetically engineered stem cells


Age-related macular degeneration (AMD) and retinitis pigmentosa (RP) are two types of eye diseases characterized by retinal degeneration that eventually leads to blindness. Gene and cell therapies have been rapidly gaining traction as potential treatment options for retinal degeneration due to the ability to specifically target the mutated gene and deliver a functional copy of that gene through a viral vector.

Researchers at the Centre for Genomic Regulation (CRG) in Barcelona are developing a stem cell treatment that focuses on two chemokine receptors, Ccr5 and Cxcr6, which they identified to be important players in cell signaling. Mesenchymal stem cells from bone marrow were modified using lentiviral vectors to have an excessive amount of Ccr5 and Cxcr6 and transplanted into models of retinal degeneration. Results reported in Molecular Journal indicate remaining function of degenerating retinal tissue was preserved.

The CRG research team will next seek to improve their Ccr5 and Cxcr6-targeted therapies, including alternative modification of the mesenchymal stem cells with adeno-associated virus (AAV) vectors, which have regulatory approval for use in gene and cell therapies.

What this means for Usher Syndrome: The modified mesenchymal stem cells with elevated levels of chemokine receptors Ccr5 and Cxcr6 resulted in improved migration and integration of the stem cells into the retina. This effectively enabled preservation of remaining retinal function.

If proven successful, this potential targeted therapy may help prevent further retinal degeneration and enable patients with AMD, RP and Usher to retain their functional vision.

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