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First Transplant using Induced Pluripotent Stem Cells: Treating Blindness

Induced pluripotent stem cells (iPSCs) have enormous potential for being used in tissue transplants and therapies. Why is this? It’s because iPSCs can be made using virtually any cells from a person’s body — such as skin or fat samples that wouldn’t be missed. (And since they’re the patient’s own cells, the immune system should not reject them in a transplant.) iPSCs are also pluripotent, which means they can be turned into nearly any cell type. This means that if a patient needs a certain type of retinal cell to treat an eye disease, a skin sample could be taken, turned into the desired retinal cells, and then transplanted into the patient’s eye. And this is exactly what happened for the first time last week.

retina age-related macular degeneration fundus
This image shows the retina of a person with intermediate age-related macular degeneration, an eye disease recently treated with iPSCs for the first time. (Image credit: National Eye Institute, National Institutes of Health)

Even though human iPSCs were first created in late 2007, it wasn’t until last week that they were used in a tissue transplant in a human for the first time. The person was a Japanese woman in her 70s, and the iPSC-derived cells were used to treat her age-related macular degeneration (AMD), a leading cause of blindness in the elderly. Safety studies in mice and monkeys had been previously done to ensure the iPSCs would not cause tumors or be rejected by the immune system.
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