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How can CRISPR-based therapies be used in research on Leber congenital amaurosis?

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CRISPR-Cas9; gene editing — gene editing The CRISPR-Cas9 gene-editing complex from the bacterium *Streptococcus pyogenes*.

CRISPR-based therapies are currently being explored in clinical trials as a potential treatment for Leber congenital amaurosis, an inherited form of vision impairment.1

In the context of research, CRISPR technology functions by utilizing a guide RNA sequence that directs a nuclease—such as the Cas9 enzyme—to a specific DNA target. By creating a precise double-stranded break at the site of a genetic defect, researchers can potentially correct the underlying mutation.213

These investigations are critical for advancing medical science, as they help researchers understand how CRISPR-induced DNA alterations affect cells, how the human immune system responds to such interventions, and how to mitigate risks associated with unwanted off-target genetic changes. While the potential to alleviate human suffering is significant, the ethical application of these technologies remains a subject of rigorous debate, emphasizing the necessity of conducting such research under strictly controlled conditions to ensure safety and efficacy.13