University of Pennsylvania : Progress toward a stem cell-based therapy for blindness.
ENPNewswire-July 29, 2022--University of Pennsylvania : Progress toward a stem cell-based therapy for blindness
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Release date- 27072022 - A multi-institutional effort led by researchers at the School of Veterinary Medicine is taking steps to develop an effective technique to regenerate photoreceptors cells and restore sight in people with vision disorders.
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. (Image: Courtesy of the Beltran laboratory/Stem Cell Reports)
What if, in people with blinding retinal disorders, one could simply introduce into the retina healthy photoreceptor cells derived in a dish from stem cells, and restore sight?
It's a tantalizingly straightforward strategy to curing blindness, yet the approach has been met with a number of scientific roadblocks, including introduced cells dying rapidly or failing to integrate with the retina.
A new study, published in Stem Cell Reports, overcomes these challenges and marks significant progress toward a cell-based therapy. The work, led by a team at the University of Pennsylvania School of Veterinary Medicine, in collaboration with researchers at the University of Wisconsin-Madison, Children's Hospital of Philadelphia, and the National Institutes of Health's National Eye Institute (NEI), introduced precursors of human photoreceptor cells into the retinas of dogs. A cocktail of immunosuppressive drugs enabled the cells to survive in the recipients' retinas for months, where they began forming connections with existing retinal cells.
'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,' says William Beltran, a professor of ophthalmology at Penn Vet and senior author on the study. 'The answer to all those questions was yes.'
Beltran and Gustavo Aguirre at Penn Vet have long been interested in addressing retinal blinding disorders and they have had great successes to date at producing corrective gene therapies for conditions with known causative genes. But for many cases of...
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