It is possible to improve vision in artisanal and unconventional ways, but science and medicine are fundamental to a definitive cure. And in this regard, genetic editing comes to the aid of those affected by hereditary blindness. At least that’s what happened to the university student Olivia Cook.
His life has always been characterized by a limited vision, similar to looking at the world through the hole of a straw. Born with a hereditary retinal disease known as amourosi congenital Of Leber or LCA, Olivia decided to participate in a clinical trial that employed the innovative gene editing tool CRISPR.
After surgery on his left eye, his vision improved significantly, particularly in low-light conditions. This therapy has shown promising results in a small cohort of patients, without causing serious side effects.
But what does this genetic editing consist of? CRISPR uses a guide RNA (gRNA) that is designed to recognize and bind to a specific DNA sequence within the genome that needs correction. Once its target sequence is found in the DNA, the Cas9 protein, an enzyme, is activated to cut both strands of the DNA at that point. The cell tries to repair the break in the DNA. During this repair process, researchers can take advantage of the cell’s natural mechanisms to insert, replace or remove segments of DNA. In many cases, the goal is correct a mutation that causes disease.
Despite the excitement, researchers remain cautious. Therapy it is not a permanent cure, but an important step forward that could slow the progression of the disease. The trial highlighted the need for further research to confirm the long-term safety of CRISPR, given that the editing system will remain in patients for life.
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