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"Retinal dystrophies often lead to blindness. Developing therapeutic interventions to restore vision is therefore of paramount importance. Here we demonstrate the ability of pluripotent stem cell‐derived cone precursors to engraft and restore light responses in the Pde6brd1 mouse, an end‐stage photoreceptor degeneration model. Our data show that up to 1.5% of precursors integrate into the host retina, differentiate into cones, and engraft in close apposition to the host bipolar cells. Half of the transplanted mice exhibited visual behavior and of these 33% showed binocular light sensitivity. The majority of retinal ganglion cells exhibited contrast‐sensitive ON, OFF or ON‐OFF light responses and even motion sensitivity; however, quite a few exhibited unusual responses (eg, light‐induced suppression), presumably reflecting remodeling of the neural retina. Our data indicate that despite relatively low engraftment yield, pluripotent stem cell‐derived cone precursors can elicit light responsiveness even at advanced degeneration stages. Further work is needed to improve engraftment yield and counteract retinal remodeling to achieve useful clinical applications."

Marcos Sotomayor, a researcher in chemistry and biochemistry at The Ohio State University, models the Usher 1F protein.

"Strategies based on the use of gene therapy to mitigate the effects of mutations that cause blindness are undergoing rapid development. Novel gene vectors now achieve widespread gene delivery and reduce the risks associated with these approaches."

"Findings are compatible with vision restoration at a visual acuity above the legal threshold for blindness defined by the World Health Organization."

"To develop a gene-agnostic therapy, we screened {approx}20 genes for their ability to prolong cone photoreceptor survival in vivo. Here, we report an adeno-associated virus (AAV) vector expressing Txnip, which prolongs the survival of cone photoreceptors and improves visual acuity in RP mouse models."

"Researchers at the Netherlands Institute for Neuroscience (NIN) have successfully delivered high-resolution implants in areas V1 and V4 of the visual cortex of monkeys, allowing the subjects to recognize artificially induced shapes. The neuroprosthetic implants consist of 1,024 electrodes. When electrical stimulation is delivered to the brain via an implanted electrode, it generates the percept of a dot of light – also known as a phosphene – at a particular location in visual space."

1024 electrons is a 32x32 electrode array - Compare that to the 64 electrode Argus 2.

"The study is the first time the safety and feasibility of adult retinal stem cell-derived RPE transplants in non-human primates was assessed. Researchers found that RPE patches transplanted into the monkey’s eye stably integrated for at least three months with no serious side effects. What is more, the stem cell-derived RPE partially took over the function of the monkey RPE and was able to support normal photoreceptor function. Importantly, these cells did not cause retinal scarring."

“We have demonstrated human cadaver donor-derived RPE at least partially replaces function in the macula of a non-human primate,” said Timothy Blenkinsop, the study’s co-lead investigator and a cellular biologist at New York City’s Icahn School of Medicine at Mount Sinai, in a statement. “Human cadaver donor-derived cells can be safely transplanted underneath the retina and replace host function, and therefore may be a promising source for rescuing vision in patients with retina diseases.”

Usher 1F Collaborative is excited to be part of this with testing set to begin in our mouse model of gene therapy developed at the University of Pittsburgh for the vision loss of Usher 1F.

"The research, led by Muna Naash, at the University of Oklahoma Health Sciences Center, with collaborators in Cleveland and Buffalo, discovered a way to deliver known gene therapies directly to the light-sensitive cells affected by this disease. "