"This isn't a cure for these diseases, but a treatment that may help people see better. This won't put back the photoreceptors that have died, but maybe give people an extra few years of useful vision with the ones that are left."
"Stem cell therapy may replace lost photoreceptors and preserve residual photoreceptors during retinal degeneration (RD)."
"It was surprisingly simple. University of California, Berkeley, scientists inserted a gene for a green-light receptor into the eyes of blind mice and, a month later, they were navigating around obstacles as easily as mice with no vision problems. They were able to see motion, brightness changes over a thousandfold range and fine detail on an iPad sufficient to distinguish letters.
"The researchers say that, within as little as three years, the gene therapy -- delivered via an inactivated virus -- could be tried in humans who've lost sight because of retinal degeneration, ideally giving them enough vision to move around and potentially restoring their ability to read or watch video."
"The most versatile treatment for inherited disorders is to precisely replace a mutated sequence with its wildtype counterpart, thereby 'normalizing' the genome. We developed a single AAV platform that allows this in retinal neurons with combined CRISPR-Cas9 and micro-homology-mediated end-joining. In blind mice, the platform rescued ~10% of the retinal neurons, resulting in an incredible ~10,000-fold improvement in light sensitivity, equivalent to the restoration mediated by conventional gene augmentation therapy."
"Researchers at the University of Science and Technology of China injected tiny nanoparticles in test mice’s eyes that bind the retina into the eyeballs, hence giving them what the team calls ‘super vision’....Moreover, scientists predict that these kinds of nanoparticles could help repair vision in humans who experience loss of retinal function or red color blindness. Also, it could prove way less invasive than the other conventional vision repair methods, and also with fewer side effects."
Stem cell therapy for RP showing promising results in early phase II clinical trial. This trial is ongoing at Massachusetts Eye and Ear in Boston.
Restore Vision 20/20 was launched as a $2.5 million initiative to drive the development of new sight-restorative treatments for retinitis pigmentosa (RP).
"The first patient in Europe to receive the Pixium Vision retinal chip implant outside of trials defines it as a “warning” system for identifying everyday objects and obstacles."
"Right now, pixels in human implants are 100 micrometers in size, and tests demonstrated that 50 micrometer pixels also work well, providing spatial resolution equivalent to about 20/200 vision. Eventually, Palanker would like to get it to 20/40 – what the state requires for a driver’s license – and the lab expects to publish a new design for achieving that resolution later this year."
Base editing is a modified version of the famous gene editing tool, CRISPR-Cas9. This next-generation technique is designed to edit single nucleotides, the building blocks of DNA, in a DNA molecule.