Researchers used CRISPR/Cas9 technology to disrupt the MYO7A gene in monkeys to create a nonhuman primate model for Usher syndrome type 1B (USH1B). Preimplantation genetic testing of embryos was used to select embryos with the desired mutation. A pregnancy was established from an embryo with the MYO7A mutation, and this infant monkey was found to have some cells that contained the desired mutation, but many cells did not show the mutation. When tested, the monkey had essentially normal hearing and vision.
What this means for Usher syndrome: This study demonstrates the feasibility of using CRISPR technology to create nonhuman primate models of Usher syndrome. Ongoing research is needed to develop models that display the symptoms that patients with Usher syndrome develop. Further advancements in this field could allow researchers to understand better the disease mechanisms and develop and test potential therapeutic interventions.
