So Brigande, Neuringer and colleagues had to genetically engineer a nonhuman primate with a gene mutation that causes Usher. However, Usher syndrome doesn’t naturally occur in nonhuman primates. A pig model of a different form of the disease, Usher Type 1C, was recently created.īut because the eyes and vision of nonhuman primates and humans are nearly identical, nonhuman primates best help scientists understand human retinal diseases and evaluate potential treatments. Scientists like Brigande already use mice to study Usher hearing loss, but fundamental differences in eye anatomy mean mice aren’t suitable models for Usher vision loss. “This model’s creation is truly a momentous scientific achievement,” Brigande said. The researchers used the gene-editing technology CRISPR/Cas9 to create the model, and thereby make it possible to test experimental gene therapies for Usher syndrome. ![]() 11 podium presentation at the Association for Research in Otolaryngology meeting. The team reported these findings during a Feb. They confirmed that their model, a rhesus macaque born a year ago, has symptoms that mirror the most severe form of Usher Syndrome, Type 1B. A treatment for Usher - which affects an estimated 4 to 17 out of every 100,000 people - has been stymied by the lack of an animal model that closely mimics how the disease affects people.Īn OHSU research team has been working to fill that gap. Genetic mutations lead those with Usher Syndrome to be born deaf, experience balance issues and gradually lose their sight. ![]() The confirmation means the research team can finally test a gene therapy for the condition, and potentially help affected children retain their sight. Those with Usher Syndrome - the leading hereditary cause for simultaneous deafness and blindness, for which there is no treatment - may have a new reason for hope, now that researchers at Oregon Health & Science University have confirmed the first-ever nonhuman primate model of their disease. ![]() OHSU researchers will now be able to put into practice the ability to correct the gene expression responsible for Usher syndrome. Usher affects an estimated 4 to 17 out of every 100,000 people.
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