Phase 1/2 trial of Brilliance showed promising results in vision improvement in people with inherited retinal disorders.
A clinical trial called Brilliance, which included 14 participants, showed that CRISPR gene editing led to improved vision in people with inherited blindness. Mass Eye and Ear researchers said their findings support further research into CRISPR therapies for inherited retinal disorders. The results of 11 participants showed improvement in vision.
Participants received an injection of the CRISPR/Cas9 genome editing drug, EDIT-101, into one eye through a specific surgical procedure. Of the 14 participants, 12 were adults, meaning they were between the ages of 17 and 63. Thus, this rare disease can be caused by more than 200 different genetic mutations. The CEP290 gene provides instructions for the production of a protein involved in the structure and function of cellular components. Therefore, mutations can lead to dysfunction and can impair the ability of photoreceptor cells to respond to light.For effectiveness, researchers looked at four criteria.
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