It has been recently announced that the first Clinical Trial will be starting to test the gene-editing technique CRISPR in the United States this Fall.

The study will focus on an inherited form of blindness (Leber congenital amaurosis) that affects 2 to 3 in about 100,000 live births. It consists of the absence of a gene function that permits the conversion of light into signals to the brain, and therefore sight itself.

This is why CRISPR has great potential: the technique consists of modifying the patient’s DNA with a “cut and edit” process that enables the replacement of the flawed gene with a healthy one. To be precise, the first gene-editing test was performed with a different tool, zinc finger nucleases, addressed to Hunter syndrome, a metabolic disease. However, this is the first Clinical Trial using a gene-editing technique, and it will consider up to 18 patients.

We are witnessing a crucial moment for this innovative therapeutic approach. Risks and potential negative implications are as yet unknown, though the CRISPR technique could really make an impact for those who are coping everyday with a rare disease without curative care.