The CRISPR gene-editing tool has been used in humans for the first time,media New Atlas reported. In a new clinical trial, scientists at Oregon Health and Science University (OHSU) are testing the technique to treat genetic mutations that cause blindness. Since its development in 2012, CRISPR has shown potential new ways to treat a range of genetic diseases. The tool allows doctors to remove problematic parts of DNA, such as those that cause disease, and can choose to replace them with beneficial substances.
The new trial, conducted at OHSU and sponsored by private companies Allergan plc and Editas Medicine, aims to treat a type of Leiber’s congenital black mon (LCA). This rare genetic disorder is triggered by a genetic mutation that affects the retina, causing the patient to become ill in early childhood and eventually leading to blindness.
For this particular type of LCA, the mutation occurs in a gene called CEP290, so this is the goal of the trial. The researchers injected the drug containing CRISPR directly into the photosensitive cells behind the retina, where they worked to remove defective genes.
CRISPR has previously been tested in the human body, but in this case, the cells are removed from the patient’ body, edited and then returned to the body. The new clinical trial marks the first time CRISPR has been working directly in the body.
Mark Pennesi, chief scientist at OHSU, said: “It is important to be able to edit genes in the human body. In addition to possibly treating previously incurable blindness, in vivo gene editing can also enable people to treat a wider range of diseases. “
The treatment is designed to be permanent to the patient, but will not be passed on to any children they may have in the future. This is an important difference because one of the main risks of gene therapy is that if something goes wrong in this situation, it could change the human gene pool from generation to generation. So far, the new CRISPR treatment has been conducted in only one patient, and it is too early to record the results. About 18 participants are expected to take part, and scientists will test the effectiveness, safety and tolerance of the treatment.