"IDIBELL researchers have used gene-editing technology in C. elegans to reproduce human mutations that cause retinitis pigmentaria. With this method, they have studied factors that affect disease development and possible drug treatments."
"Identification of GPR108 as 'lock' for adeno-associated virus vector (AAV) is crucial to this emerging novel class of genetic medicine and may provide framework for developing more targeted gene therapies."
Although the lens hadn’t been launched commercially it is considered as a breakthrough lens by the FDA or the Food and Drug Administration in the US. People with different visual ailments like retinitis pigmentosa or macular degeneration will be benefited the most. Mojo Vision had raised over $100 million with this project. Executives from Apple Inc., Google, and other tech giants have been working with the startup along with several opticians and ophthalmologists.
Because semiconductive polymers bend and flex like natural tissues, Lanzani says, 'they are biocompatible.' In tests in the lab and in animals, the polymer retina seems to coexist with them quite happily, with no adverse reactions at all.
"Just as important, adds IIT neuroscientist Fabio Benfenati, semiconducting polymers can get the physiology right. When light hits the polymer sheet, he says, it triggers a localized pulse of electrical activity about 80 to 100 micrometers across. Because this is roughly comparable to the spacing of rod and cone cells outside the densely packed fovea, where the eye’s visual acuity is the highest, the polymer prosthesis would allow a resolution akin to a person’s natural peripheral vision."
Just as gene therapy finally seems to be living up to its promise, a study has revived a lingering worry about the viral vessel that many efforts rely on to ferry therapeutic genes into patients. This “vector,” a stripped-down version of adeno-associated virus (AAV), was thought to be safe because it rarely knits its cargo of human DNA into a cell’s chromosomes, where it might activate cancer-causing genes. But a study of dogs with hemophilia, treated with AAV up to 10 years earlier, has shown that the vector can readily insert its payload into the host’s DNA near genes that control cell growth.
"The authors note that these data indicate that emixustat can reduce metabolic and oxygen demands in the retina under dark conditions, which has implications for the treatment of ischemic retinal diseases in which hypoxia plays a prominent role."
"The safety profile is strong and that the number of patients involved is continuing to grow. More good news is that despite using an allogeneic approach, immune suppression doesn’t seem necessary for engraftment."
"Researchers at the National Eye Institute (NEI) are launching a clinical trial to test the safety of a novel patient-specific stem cell-based therapy to treat geographic atrophy, the advanced “dry” form of age-related macular degeneration (AMD), a leading cause of vision loss among people age 65 and older. The geographic atrophy form of AMD currently has no treatment.
“The protocol, which prevented blindness in animal models, is the first clinical trial in the U.S. to use replacement tissues from patient-derived induced pluripotent stem cells."
"Here, we provide a summary of these emerging data and a discussion of technical concerns specific to AAV-mediated reprogramming experiments that must be addressed in order for the field to move forward."
"Existing retinal prostheses require silicon or metal implants that are comparatively thick and completely rigid, a combination that the sensitive retinal tissue does not like at all...So instead, ...their group is investigating a different kind of retinal prosthesis made from semiconductive polymers, a class of carbon-based plastics that can conduct electricity in much the same way that silicon microchips do...Because semiconductive polymers bend and flex like natural tissues, Lanzani says, “they are biocompatible.” In tests in the lab and in animals, the polymer retina seems to coexist with them quite happily, with no adverse reactions at all."