The first clinical trial involving the use of the gene editing tool CRISPR directly in the patient (see more ) has been approved in the United States. The trial aim to treat a congenital blindeness will include around 15 patients, adults and children, in whom the therapy will be applied in one of their eyes.

CRISPR has been previously used in humans, but in the so-called “ex vivo” modality. This consists of modifying the genome of cells extracted from the patient before returning the modified cells to their body. Now, this trial goes one step further, proposing the use of the tool directly in the patient.

The aim is to treat a condition that causes hereditary blindness, called Leber Congenital Amaurosis. This disease is usually caused by a mutation in the CEP290 gene, which is it hoped to correct using CRISPR.

Ethical concerns

From an ethical point of view, this application of gene editing is framed within somatic gene editing, which unlike germline gene editing (in sperm, eggs and embryos), does not involve the manipulation and destruction of human embryos, nor the transmission of genetic changes to offspring. Nevertheless, there are still safety risks associated with the technique (see HERE and HERE), so that clinical trials must be conducted with the utmost care, even more, if this practice is directly performed in the patient’s own body.


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