Successful treatment of gene therapy for the blind

US researchers said a year ago, three blind people in their 20s volunteered to treat with gene therapy. Now, their eyesight has improved significantly, even a sick person can read the numbers of the car speedometer. Relevant information will be published in the New England Journal of Medicine on August 23.

These three volunteers suffered from congenital blindness (Leber Congenital Amaurosis) , a type of genetic retinopathy caused by a gene called RPE65 (the RPE65 gene produces a special type of Vitamin A for sagging cells). In vivo) mutations occur that make the patient's eyesight decrease with age. So some people after their adulthood were blind, there is currently no effective treatment for this disease.

The team of Artur V. Cideciyan of the University of Pennsylvania injected normal DNA into the virus, then reintroduced the virus into the patient's eye cells . After 3 months, the eyesight of 3 volunteers has been significantly improved, visible light can be seen with the naked eye, something they could not do before. Moreover, after 1 year of treatment, their eyes and health did not occur any immune response.

In particular, a female patient during treatment created ' second vision ', her brain learned after gene therapy treatment was able to recover the search and gather information in one certain area of ​​the retina, making her read the numbers on the car speedometer.

In 1999, an 18-year-old girl who suffered from liver disease after 4-day treatment with gene therapy died, this event caused gene therapy to face a lot of criticism and made people more suspicious Gene therapy.

Artur V. Cideciyan said: ' RPE65 gene therapy is very safe, the patient's vision is markedly improved, hoping to continue conducting experiments to address safety and calculation issues. effective '.

Congenital blindness is a variable retinal pigment, in the United States there are about 2000 people with congenital blindness; 200,000 people suffer from modified retinal pigment.