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Research

Rescued photoreceptors prevent blindness

05/03/2019
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After success in animal models, researchers believe they have paved the way for the first-ever human clinical trials of a stem cell based therapy for atrophic age-related macular degeneration (AMD).

According to researchers at the US National Eye Institute (NEI), the novel patient-specific stem cell-based therapy successfully prevented blindness in animal models of atrophic AMD.

The technique involves taking a patient’s blood cells and converting them into induced pluripotent stem cells (iPSC), which can become any type of cell in the body.

The iPSC’s are programmed to become retinal pigment epithelial (RPE) cells, the ones that die early in the geographic atrophy stage of macular degeneration.

In geographic atrophy, when RPE cells die photoreceptors also expire, resulting in blindness. The therapy is an attempt to revive the health of remaining photoreceptors by replacing dying RPE cells with iPSC-derived RPE cells.

Before transplantation, the iPSC-derived RPE cells are grown in sheets as thin as a single cell, replicating their natural structure within the eye. This monolayer of iPSC-derived RPE cells is grown on a biodegradable scaffold designed to promote the integration of the cells within the retina.

The team studied the method in rat and pig models. Ten weeks after the human iPSC-derived RPE patches were implanted in the animals’ retinas, imaging studies confirmed that the lab-made cells had integrated within the animal retina

“If the clinical trial moves forward, it would be the first ever to test a stem cell-based therapy derived from induced pluripotent stem cells for treating a disease,” lead investigator Prof Kapil Bharti, head of the National Institutes for Health NEI Unit on Ocular and Stem Cell Translational Research, said.

The results were published in the journal Science Translational Medicine.

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