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GEN News Highlights : Jul 21, 2013

Seeing the Light: Mature Photoreceptors Made from Stem Cells

It has been a big month for regenerative medicine, with one group of researchers reporting on their generation of human induced pluripotent stem cell (iPSC)-derived liver buds, and another presenting an approach for the generation of human iPSC-derived vascular networks. As part of both of the described studies, scientists reported having successfully transplanted their lab-grown structures into mice.

Today, researchers at University College London and colleagues report their generation of light-sensitive photoreceptor cells from murine embryonic stem cells, which they’ve also successfully transplanted into mice.

The London-based team, led by Robin Ali, Ph.D., presents in Nature Biotechnology an overview of their approach, and show that these lab-grown light-sensing cells can integrate into the retinas of adult mice with eye disease.

Age-related macular degeneration, Dr. Ali et al. note, is but one inherited disorder that causes the function of the retina to be lost through photoreceptor damage.

By growing replacement photoreceptors in the lab, researchers envision one day treating patients with retinal disease using stem cells.

“Until recently, the complex structure of the retina has proved difficult to reproduce in the lab. This is probably because the type of cell culture we were using was not able to recreate the developmental process that would happen in a normal embryo,” Dr. Ali said in a statement. The 3D technique his team employed “more closely mimics normal development, which means we are able to pick out and purify the cells at precisely the right stage to ensure successful transplantation,” he continued.

Once transplanted, the lab-grown photoreceptors matured into cells that seem much like fully developed, functional rod photoreceptors, the researchers note. Because doing so would require many more transplantable cells than could be produced for this study, the researchers did not assess whether the recipient mice showed improved vision. Among other next steps, Dr. Ali said one future effort “will be to refine this technique using human cells to enable us to start clinical trials.”

Commenting on the study, Rob Buckle, Ph.D., head of regenerative medicine at the Medical Research Council—which funded the work in part—called the achievement of Dr. Ali et al. an “important milestone on the road to developing a widely available cell therapy for blindness.”

"Photoreceptor precursors derived from threedimensional embryonic stem cell cultures integrate and mature within adult degenerate retina" was published July 21 in Nature Biotechnology.