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In this study, two guide RNAs and S. aureus Cas9 expressed under the control of a photoreceptor-specific promoter were delivered in a single, subretinally-injected adeno-associated virus (AAV) at two different doses. Retinal tissue and genomic DNA were taken from within the sub-macular bleb region at six and 13 weeks and analyzed using Editas Medicine’s novel sequencing method, UDiTaS™, to accurately quantify all editing events. Gene editing was demonstrated to be dose and time dependent. Editing rates were measured directly as a percentage of all alleles in total genomic DNA taken from the retinas of non-human primates, which included both photoreceptor cells and other cell types. Editing rates were also projected based on additional analyses which demonstrated that Cas9 expression was limited to photoreceptor cells, which are the target cells for LCA10 treatment. For animals treated with the higher dose, the projected productive editing rate may be as high as 50 percent in photoreceptor cells, based on directly measured editing of 15 percent in total genomic DNA and an estimate for the proportion of cells represented by photoreceptors. Achieving 50 percent of all alleles would be well above the editing rate hypothesized to have a therapeutic effect in patients.
“Based on analyses presented today, we believe that we were successful in editing the vast majority photoreceptors using a primate-specific candidate that shares critical design and construction features with our clinical candidate,” said
About Leber Congenital Amaurosis 10
Leber Congenital Amaurosis, or LCA, is a group of inherited retinal dystrophies caused by mutations in at least 18 different genes. It is the most common cause of inherited childhood blindness, with an incidence of two to three per 100,000 live births worldwide. Symptoms of LCA appear within the first year of life, resulting in significant vision loss and blindness. The most common form of the disease, referred to as LCA10, is a monogenic disorder caused by mutations in the CEP290 gene and represents approximately 20‑30 percent of all LCA subtypes.
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