CRISPR vs. Leber's Amaurosis
The CRISPR genetic editing technology directly in the patient's body was used for the first time to treat blindness in humans
Polina Gershberg, Naked Science
Scientists have announced the first use of the CRISPR gene editing tool inside someone's body to treat blindness. According to the researchers, this was the taking of a new frontier in the treatment of diseases. An article published by the Associated Press indicates that the patient recently underwent such an intervention at the Casey Eye Institute of Oregon Health and Science University in Portland. The reason for the need for treatment was a hereditary form of blindness. It is emphasized that details about the patient or when exactly the operation took place will not be reported.
A new method is being tested for the treatment of a congenital disease – Leber's amaurosis. It is caused by a gene mutation that prevents the body from producing the protein needed to convert light into signals for the brain that provides vision. People with this disease are often born with a weak ability to see and may even lose it within just a few years.
Conventional gene therapy – substitution of a replacement gene – will not work, because with it the right gene carries the virus, and the defective gene in such patients is too large. Therefore, the only way is to edit or remove the damaged area by cutting the DNA. That is why CRISPR technology was chosen, and all manipulations are performed on the patient's retina under general anesthesia.
It takes about a month to make it clear how working the vision restoration technique will be. If the first few attempts are safe, the doctors plan to continue the tests by performing the necessary manipulations in 18 more people. Other medical institutions have also already planned to participate in the study. Dr. Jason Komander, an eye surgeon at one of them, the Massachusetts Eye and Ear Hospital in Boston, notes that this marks a "new era in medicine" using technology that "makes DNA editing much easier and more efficient."
"We have the potential to take people who are essentially blind and make them see," says Charles Albright, chief scientist at Editas Medicine (the company developing this treatment). According to doctors, the eye surgery itself poses a small risk: infections and bleeding are relatively rare complications.
According to Dr. Eric Pierce, who is not involved in the study, one of the biggest potential risks from gene editing is that CRISPR can make unintended changes to other, neighboring genes, however, scientists have made every effort to minimize the likelihood of this.
Dr. Kiran Musunuru from the University of Pennsylvania, an expert in gene editing, notes that the treatment should work: the technique was successful in animal and human tissue testing. The gene editing tool, Dr. Musunuru notes, remains in sight and does not move to other parts of the body, so "if something goes wrong, the probability of harm is very small; this is a good first step for editing genes in the body."
It is difficult to overestimate the importance of new methods of treating such diseases. Today, people with many diseases simply have no chance of improving their condition. New methods can advance the development of therapy in many directions at once, not only for eye diseases. CRISPR is the basis of research that is currently at various stages, for editing both inside the body (for example, for the treatment of metabolic diseases) and outside it (for example, for the treatment of cancer and sickle cell anemia).
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