Sneha Khedkar is a biologist-turned-freelance-science-journalist from India. She holds a master's degree in biochemistry and a bachelor's degree in microbiology and biochemistry. After her master's, she worked as a research fellow for four years, studying stem cell biology.
A CRISPR therapy injected directly into the eye shows promise in treating the most common form of inherited vision loss in children, an early trial suggests.
LCA currently has no cure — but now, there's evidence that the famous CRISPR gene-editing tool could be safely used to improve the vision of some people with the condition. The results of the early-stage trial were published May 6 in The New England Journal of Medicine. By submitting your information you agree to the Terms & Conditions and Privacy Policy and are aged 16 or over.The trial is also notable in that it included the first person to ever receive a CRISPR-based treatment directly into the body. By comparison, the first approved CRISPR therapy involves removing cells from the body, editing them in a lab and then returning them to the patient.
The team used a CRISPR-based strategy because CEP290 is a large gene, making it a difficult target for conventional gene therapy, Pennesi said. Some gene therapies use modified viruses to deliver functional genes into cells, to replace faulty genes, but the CEP290 gene is too large to fit into such a delivery system.
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