An inherited form of blindness might be cured thanks to CRISPR, a gene-editing technique that works from inside the body. This technique will begin being tested for the first time in a study that waits for the patients to be enrolled.
What people will be treated, and how does this work?
The eyes of these people are not abnormal, but the brain can’t enable sight because it does not get the signal. These people are born without the gene that converts light into signals. The goal of the experimental treatment is to offer a healthy version of the gene to adults and kids that do not have it. In order to do so, the scientists will use a tool that edits the DNA where it needs to be modified. The native DNA of the person is altered irrevocably, so this treatment can’t happen more than once.
Almost 18 people will be tested by Allergan and Editas Medicine, the testing companies starting in autumn. Patients are also coming from Massachusetts Eye and Ear in Boston. Gene editing inside the body has been tried one more time by Sangamo Therapeutics using a tool called zinc fingers to treat metabolic diseases.
Another version of the CRISPR gene-editing technology
A Chinese scientist proposed a more controversial technique last year. They thought of altering the DNA of embryos at conception in such a way that future generation will not suffer from the same disease. Unfortunately, the DNA changes in the US study will not be passed on to offsprings
A gene-editing tool like CRISPR can be so easy to use, and this is what captivates scientists, that they can help patients faster. However, they do not know all the risks yet. The chances for this to be useful in curing inherited blindness are more significant than the risks.