The first edit of genes in the human body: CRISPR cure hereditary blindness?

editing Tool CRISPR genes first used directly in the human body.

Scientists from the University of Oregon science and health applied this technology to treat a rare genetic disease Leber Ambrose. With this ailment in the retina die and do not regenerate light-sensitive cells. As a result, patients are either born blind or lose their sight within the first few years of life.

the Specialists explained that in this case the patient had a mutation in the gene CEP290. It had to be removed, cutting the DNA on both sides of the mutation. Subsequently, the ends of DNA strands had to come together, and once a defective gene had to earn it.

to Replace a gene on the “right” scientists, it was not possible, since viral particles that are usually used for such manipulations, this gene is too large.

the Procedure was performed under General anesthesia in an hour. Professionals were introduced a CRISPR-drug directly in photoreceptor cells of the retina.

Now the patient is under observation. The results of treatment so far. It can take up to a month to evaluate how effective the technique, the Associated Press reports. Thus, when the surgery was performed, doctors did not specify.

CRISPR-drug was administered via subretinal injection.OHSU photo/Kristyna Wentz-Graff.

the Study was sponsored by pharmaceutical companies, Allergan and Editas Medicine. They developed an experimental facility for subretinal injection.

On the website Editas Medicine reported that if the results of the first operation will be satisfactory in the first phase of clinical trials of a new drug to take by 18 patients. This will not only adults but also children and adolescents (aged 3 to 17 years). They were divided into several groups, in which Lecarstvo will be administered in a different dosage.

Doctors say that the risks associated with the surgery itself, minimum: infection and bleeding are relatively rare complications.

Much more geneticists concerned about unintended changes to other genes. However, the developers of the product have done everything possible to avoid this “side effect”.

Experts hope that clinical trials will confirm the efficacy and safety of the experimental procedure. Then the doctors will be able to save people from diseases that were not previously respond to treatment. In this case, after manipulation, the disease will not be passed on to children patients inherited.

Experts stress that the new clinical trial was a turning point in the history of CRISPR-therapy, in fact, repeat this procedure for genome editing was first carried out in vivo, i.e. directly in the human body.

Scientists hope that in the future these operations they will be able to treat many different diseases.

Recall that in 2017, scientists for the first time in the history of medicine, edited by gene directly into the human body using a different technique. Later, the researchers presented the first results of the breakthrough work that “Conduct.Science” (nauka.vesti.ru) told in detail.

Text: To.Science