Edwin Stone, MD, PhD, University of Iowa Institute for Vision Research, talks about curing inherited retinal diseases.
"Our data show that fetal retinal sheet transplants can result in remarkably normal visual function in visual cortex of rats with a degenerated host retina and represents a critical step towards developing an effective remedy for the visually impaired human population."
Eyes are ‘leading the way’
A leading candidate for stem cell therapies is blindness caused by disorders such as retinitis pigmentosa and macular degeneration.
Injecting an experimental treatment into a blind eye carries a relatively low risk, said Gamm, a pediatric ophthalmologist. Eyes are encapsulated, so wayward cells likely wouldn’t travel to other parts of the body. If something goes wrong, an eye can be removed. Doctors wanting to see how transplanted cells are behaving can dilate the pupils and look — no MRI or PET scan required.
“The eye is leading the way,” said Gamm, who noted that many of the human trials of stem cell therapies taking place today are treatments for vision disorders.
"GS030 is based on the Optogenetics technology platform developed by GenSight, which uses gene therapy to introduce a gene encoding for a light-sensitive protein into retinal ganglion cells by a single intravitreal injection, thereby making them responsive to light and bypassing destroyed photoreceptors."
Researchers at MIT have discovered a Cas9 enzyme that can target almost half of the locations on the genome, significantly widening its potential use.
"Today, the Foundation Fighting Blindness announced the creation of the first-ever Retinal Degeneration Fund (RD Fund), a wholly owned, 501(c)(3) not-for-profit subsidiary focused on making venture philanthropy investments to further the Foundation's mission: To provide preventions, treatments and cures for inherited retinal diseases and age-related macular degeneration as fast as possible. The RD Fund, which now has more than $70 million in initial funding, will invest in companies with projects nearing clinical testing."
"A team of Chinese researchers have sparked hope of a treatment for degenerative eye diseases, developing nanowires which can be implanted in the eye and restore sight...More excitingly though, in the brains of the mice the scientists could detect light-evoked activity in the primary visual cortex. This, they say, confirms the restoration of visual function just 4–8 weeks after implant surgery."
Jack was born with an eye condition known as retinitis pigmentosa associated with RPE65 gene mutations. Two months ago, he was treated with a newly FDA-approved gene therapy drug known as Luxturna — and the results are life-changing.
“Mom, that was the best day ever,” Jack Hogan said when he noticed he could see the whiteboard in his classroom for the very first time without his visual aid.
“Researchers have reversed congenital blindness in mice by changing supportive cells in the retina called Muller glia into rod photoreceptors. The findings advance efforts toward regenerative therapies for blinding diseases such as age-related macular degeneration and retinitis pigmentosa.”
The Australian government is funding research for a cure for the vision loss of Usher 1F.