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Progress toward a stem cell–based therapy for blindness

July 28, 2022

Collaborators from the University of Pennsylvania, University of Wisconsin, Children’s Hospital of Philadelphia, and NEI's Intramural Research Program, successfully implanted human-derived photoreceptor precursor cells into the retinas of dogs. Photoreceptors are light-sensing cells in the retina that enable vision. Dozens of rare eye conditions cause the loss of photoreceptor cells - common ones, too, including age-related macular degeneration. Photoreceptors communicate with other retinal neurons that in turn signal the brain. The project was funded by NEI's Audacious Goals Initiative, an effort to restore vision through regeneration of the retina.

Photoreceptor precursors

Following a transplantation procedure, human photoreceptor precursor cells, labeled red, migrated and integrated into a degenerated canine retina. The green label is a synaptic maker, suggesting the transplanted cells began forming a connection with second-order neurons in the retina. Credit: William Beltran, U. Penn.

“In this study, we wanted to know if we could, one, improve the surgical delivery of these cells to the subretinal space; two, image the cells in vivo; three, improve their survival; and four, see them migrate to the layer of the retina where they should be and start integrating,” said William Beltran, a professor of ophthalmology at the University of Pennsylvania School of Veterinary Medicine and senior author on the study. “The answer to all those questions was yes.”

University of Pennsylvania news story