UCI researcher receives CIRM funding for stem cell-based retina therapy to treat blindness
UNIVERSITY OF CALIFORNIA – IRVINE
University of California, Irvine stem cell researcher Magdalene J. Seiler, PhD, has received a $4.8 million grant from the California Institute of Regenerative Medicine (CIRM) to continue developing a stem cell-based therapy for a vision-robbing eye disease, retinitis pigmentosa. The therapy may also be applicable to macular degeneration.
“Our goal is a treatment based on transplanting sheets of stem-cell derived retina, called retina organoids, to the back of the eye,” said Seiler, an assistant professor in the Department of Physical Medicine & Rehabilitation, UCI School of Medicine, and a researcher with the Sue & Bill Gross Stem Cell Research Center. “Advanced stages of this disease show irreversible loss of photoreceptors, the light-sensing cells in the back of the eye, and patients gradually go blind.”
Retinitis pigmentosa and age-related macular degeneration lead to the incurable loss of vision in millions of people. No traditional therapy can restore advanced stages of retinal degeneration due to the irrevocable loss of photoreceptors. While retinitis pigmentosa (RP) is designated an orphan disease by the FDA, age-related macular degeneration (AMD) is among the leading causes of blindness in adults over age 65.
Since 1995, Seiler and her team have pursued promising research into the development and use of retinal sheet transplantation, using a unique patented implantation instrument and procedure which has demonstrated improvement in visual responses in four different retinal degeneration models.
“The aim is to replace damaged photoreceptors with the hope of re-establishing neural circuitry within the eye,” Seiler said.
In 2014, Seiler and colleagues received a CIRM grant to develop human embryonic stem cells for transplantable 3-D sheets of retinal cells for vision restoration. Their work, published in Investigative Ophthalmology & Visual Science, demonstrated that stem-cell derived retina can improve visual responses in two different immunodeficient rat models with retinal degeneration that do not reject human cells…..